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UF Health University of Florida
Department of Ophthalmology Research College of Medicine
Ocular Gene Therapy Core Overview

Vector Utilization History

AAV capsid structure

The AAV vectors produced by the gene therapy vector core at the University of Florida have been used in hundreds of studies of ocular disease, ocular biology, and to establish pre-clinical efficacy and safety. The following bibliography identifies some of the studies using vectors produced at the University of Florida:

  1. Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs. Boyd RF, Sledge DG, Boye SL, Boye SE, Hauswirth WW, Komáromy AM, Petersen-Jones SM, Bartoe JT. Gene Ther. 2016 Apr;23(4):400. doi: 10.1038/gt.2016.10. No abstract available. PMID: 27052928 [PubMed – in process].
  2. Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs. Boyd RF, Boye SL, Conlon TJ, Erger KE, Sledge DG, Langohr IM, Hauswirth WW, Komáromy AM, Boye SE, Petersen-Jones SM, Bartoe JT. Gene Ther. 2016 Apr 7. doi: 10.1038/gt.2016.31. [Epub ahead of print]. PMID: 27052802 [PubMed – as supplied by publisher].
  3. Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia. Ye GJ, Budzynski E, Sonnentag P, Nork TM, Miller PE, Sharma AK, Ver Hoeve JN, Smith LM, Arndt T, Calcedo R, Gaskin C, Robinson PM, Knop DR, Hauswirth WW, Chulay JD. Hum Gene Ther Clin Dev. 2016 Mar;27(1):37-48. doi: 10.1089/humc.2015.164. PMID: 27003753 [PubMed – in process].
  4. Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia. Ye GJ, Budzynski E, Sonnentag P, Nork TM, Miller PE, McPherson L, Ver Hoeve JN, Smith LM, Arndt T, Mandapati S, Robinson PM, Calcedo R, Knop DR, Hauswirth WW, Chulay JD. Hum Gene Ther Clin Dev. 2016 Mar;27(1):27-36. doi: 10.1089/humc.2015.163. PMID: 27003752 [PubMed – in process].
  5. AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy. Dinculescu A, Stupay RM, Deng WT, Dyka FM, Min SH, Boye SL, Chiodo VA, Abrahan CE, Zhu P, Li Q, Strettoi E, Novelli E, Nagel-Wolfrum K, Wolfrum U, Smith WC, Hauswirth WW. PLoS One. 2016 Feb 16;11(2):e0148874. doi: 10.1371/journal.pone.0148874. eCollection 2016. PMID: 26881841 [PubMed – in process] Free PMC Article. 
  6. Impact of Heparan Sulfate Binding on Transduction of Retina by Recombinant Adeno-Associated Virus Vectors. Boye SL, Bennett A, Scalabrino ML, McCullough KT, Van Vliet K, Choudhury S, Ruan Q, Peterson J, Agbandje-McKenna M, Boye SE. J Virol. 2016 Mar 28;90(8):4215-31. doi: 10.1128/JVI.00200-16. Print 2016 Apr 15. PMID: 26865709 [PubMed – in process].
  7. Targeting the Nrf2 Signaling Pathway in the Retina With a Gene-Delivered Secretable and Cell-Penetrating Peptide. Ildefonso CJ, Jaime H, Brown EE, Iwata RL, Ahmed CM, Massengill MT, Biswal MR, Boye SE, Hauswirth WW, Ash JD, Li Q, Lewin AS. Invest Ophthalmol Vis Sci. 2016 Feb 1;57(2):372-86. doi: 10.1167/iovs.15-17703. PMID: 26842755 [PubMed – in process].
  8. Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial. Ghazi NG, Abboud EB, Nowilaty SR, Alkuraya H, Alhommadi A, Cai H, Hou R, Deng WT, Boye SL, Almaghamsi A, Al Saikhan F, Al-Dhibi H, Birch D, Chung C, Colak D, LaVail MM, Vollrath D, Erger K, Wang W, Conlon T, Zhang K, Hauswirth W, Alkuraya FS. Hum Genet. 2016 Mar;135(3):327-43. doi: 10.1007/s00439-016-1637-y. Epub 2016 Jan 29. PMID: 26825853 [PubMed – in process].
  9. Healthy and diseased corticospinal motor neurons are selectively transduced upon direct AAV2-2 injection into the motor cortex. Jara JH, Stanford MJ, Zhu Y, Tu M, Hauswirth WW, Bohn MC, DeVries SH, Özdinler PH. Gene Ther. 2016 Mar;23(3):272-82. doi: 10.1038/gt.2015.112. Epub 2016 Jan 21. PMID: 26704722 [PubMed – in process] Free PMC Article. 
  10. Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma? Berns KI, Byrne BJ, Flotte TR, Gao G, Hauswirth WW, Herzog RW, Muzyczka N, VandenDriessche T, Xiao X, Zolotukhin S, Srivastava A. Hum Gene Ther. 2015 Dec;26(12):779-81. doi: 10.1089/hum.2015.29014.kib. No abstract available. PMID: 26690810 [PubMed – indexed for MEDLINE].
  11. Cone-Specific Promoters for Gene Therapy of Achromatopsia and Other Retinal Diseases. Ye GJ, Budzynski E, Sonnentag P, Nork TM, Sheibani N, Gurel Z, Boye SL, Peterson JJ, Boye SE, Hauswirth WW, Chulay JD. Hum Gene Ther. 2016 Jan;27(1):72-82. doi: 10.1089/hum.2015.130. PMID: 26603570 [PubMed – in process].
  12. Pathological Effects of Mutant C1QTNF5 (S163R) Expression in Murine Retinal Pigment Epithelium. Dinculescu A, Min SH, Dyka FM, Deng WT, Stupay RM, Chiodo V, Smith WC, Hauswirth WW. Invest Ophthalmol Vis Sci. 2015 Oct;56(11):6971-80. doi: 10.1167/iovs.15-17166. PMID: 26513502 [PubMed – indexed for MEDLINE] Free PMC Article.  
  13. Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs. Boyd RF, Sledge DG, Boye SL, Boye SE, Hauswirth WW, Komáromy AM, Petersen-Jones SM, Bartoe JT. Gene Ther. 2016 Feb;23(2):223-30. doi: 10.1038/gt.2015.96. Epub 2015 Oct 15. PMID: 26467396 [PubMed – in process] Free PMC Article. 
  14. Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease. Beltran WA, Cideciyan AV, Iwabe S, Swider M, Kosyk MS, McDaid K, Martynyuk I, Ying GS, Shaffer J, Deng WT, Boye SL, Lewin AS, Hauswirth WW, Jacobson SG, Aguirre GD. Proc Natl Acad Sci U S A. 2015 Oct 27;112(43):E5844-53. doi: 10.1073/pnas.1509914112. Epub 2015 Oct 12. PMID: 26460017 [PubMed – indexed for MEDLINE] Free Article.
  15. Consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice. Yu H, Koilkonda RD, Chou TH, Porciatti V, Mehta A, Hentall ID, Chiodo VA, Boye SL, Hauswirth WW, Lewin AS, Guy J. Proc Natl Acad Sci U S A. 2015 Oct 20;112(42):E5689-98. doi: 10.1073/pnas.1506129112. Epub 2015 Oct 5. PMID: 26438859 [PubMed – indexed for MEDLINE] Free PMC Article.
  16. Characterization of Ribozymes Targeting a Congenital Night Blindness Mutation in Rhodopsin Mutation. Conley SM, Whalen P, Lewin AS, Naash MI. Adv Exp Med Biol. 2016;854:509-15. doi: 10.1007/978-3-319-17121-0_68. PMID: 26427453 [PubMed – indexed for MEDLINE].
  17. Gene Therapy for MERTK-Associated Retinal Degenerations. LaVail MM, Yasumura D, Matthes MT, Yang H, Hauswirth WW, Deng WT, Vollrath D. Adv Exp Med Biol. 2016;854:487-93. doi: 10.1007/978-3-319-17121-0_65. PMID: 26427450 [PubMed – indexed for MEDLINE].
  18. A Mini-review: Animal Models of GUCY2D Leber Congenital Amaurosis (LCA1). Boye SE. Adv Exp Med Biol. 2016;854:253-8. doi: 10.1007/978-3-319-17121-0_34. Review. PMID: 26427419 [PubMed – indexed for MEDLINE].
  19. Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection. Lipinski DM, Reid CA, Boye SL, Peterson JJ, Qi X, Boye SE, Boulton ME, Hauswirth WW. Hum Gene Ther. 2015 Nov;26(11):767-76. doi: 10.1089/hum.2015.097. Epub 2015 Sep 29. PMID: 26359319 [PubMed – in process].
  20. Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness. Scalabrino ML, Boye SL, Fransen KM, Noel JM, Dyka FM, Min SH, Ruan Q, De Leeuw CN, Simpson EM, Gregg RG, McCall MA, Peachey NS, Boye SE. Hum Mol Genet. 2015 Nov 1;24(21):6229-39. doi: 10.1093/hmg/ddv341. Epub 2015 Aug 26. PMID: 26310623 [PubMed – in process].
  21. Gene Therapy Fully Restores Vision to the All-Cone Nrl(-/-) Gucy2e(-/-) Mouse Model of Leber Congenital Amaurosis-1. Boye SL, Peterson JJ, Choudhury S, Min SH, Ruan Q, McCullough KT, Zhang Z, Olshevskaya EV, Peshenko IV, Hauswirth WW, Ding XQ, Dizhoor AM, Boye SE. Hum Gene Ther. 2015 Sep;26(9):575-92. doi: 10.1089/hum.2015.053. Epub 2015 Aug 6. PMID: 26247368 [PubMed – in process] Free PMC Article. 
  22. Improvement in vision: a new goal for treatment of hereditary retinal degenerations. Jacobson SG, Cideciyan AV, Aguirre GD, Roman AJ, Sumaroka A, Hauswirth WW, Palczewski K. Expert Opin Orphan Drugs. 2015 May 4;3(5):563-575.  PMID: 26246977 [PubMed] Free PMC Article. 
  23. Kinesin family 17 (osmotic avoidance abnormal-3) is dispensable for photoreceptor morphology and function. Jiang L, Tam BM, Ying G, Wu S, Hauswirth WW, Frederick JM, Moritz OL, Baehr W. FASEB J. 2015 Dec;29(12):4866-80. doi: 10.1096/fj.15-275677. Epub 2015 Jul 30. PMID: 26229057 [PubMed – in process].
  24. Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia. Banin E, Gootwine E, Obolensky A, Ezra-Elia R, Ejzenberg A, Zelinger L, Honig H, Rosov A, Yamin E, Sharon D, Averbukh E, Hauswirth WW, Ofri R.  Mol Ther. 2015 Sep;23(9):1423-33. doi: 10.1038/mt.2015.114. Epub 2015 Jun 19. PMID: 26087757 [PubMed – in process].
  25. Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-Linked Retinitis Pigmentosa. Deng WT, Dyka FM, Dinculescu A, Li J, Zhu P, Chiodo VA, Boye SL, Conlon TJ, Erger K, Cossette T, Hauswirth WW. Hum Gene Ther. 2015 Sep;26(9):593-602. doi: 10.1089/hum.2015.035. Epub 2015 Jul 29. PMID: 26076799 [PubMed – in process].
  26. Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and Rat. Bogner B, Boye SL, Min SH, Peterson JJ, Ruan Q, Zhang Z, Reitsamer HA, Hauswirth WW, Boye SE. PLoS One. 2015 Jun 8;10(6):e0128759. doi: 10.1371/journal.pone.0128759. eCollection 2015. PMID: 26052939 [PubMed – indexed for MEDLINE] Free PMC Article. 
  27. AAV8(Y733F)-mediated gene therapy in a Spata7 knockout mouse model of Leber congenital amaurosis and retinitis pigmentosa. Zhong H, Eblimit A, Moayedi Y, Boye SL, Chiodo VA, Chen Y, Li Y, Nichols RM, Hauswirth WW, Chen R, Mardon G. Gene Ther. 2015 Aug;22(8):619-27. doi: 10.1038/gt.2015.42. Epub 2015 May 12. PMID: 25965394 [PubMed – indexed for MEDLINE].
  28. Improvement and decline in vision with gene therapy in childhood blindness. Jacobson SG, Cideciyan AV, Roman AJ, Sumaroka A, Schwartz SB, Heon E, Hauswirth WW. N Engl J Med. 2015 May 14;372(20):1920-6. doi: 10.1056/NEJMoa1412965. Epub 2015 May 3. PMID: 25936984 [PubMed – indexed for MEDLINE] Free PMC Article.
  29. Vitreal delivery of AAV vectored Cnga3 restores cone function in CNGA3-/-/Nrl-/- mice, an all-cone model of CNGA3 achromatopsia. Du W, Tao Y, Deng WT, Zhu P, Li J, Dai X, Zhang Y, Shi W, Liu X, Chiodo VA, Ding XQ, Zhao C, Michalakis S, Biel M, Zhang Z, Qu J, Hauswirth WW, Pang JJ. Hum Mol Genet. 2015 Jul 1;24(13):3699-707. doi: 10.1093/hmg/ddv114. Epub 2015 Apr 8. PMID: 25855802 [PubMed – in process].
  30. Gene therapy with the caspase activation and recruitment domain reduces the ocular inflammatory response. Ildefonso CJ, Jaime H, Biswal MR, Boye SE, Li Q, Hauswirth WW, Lewin AS. Mol Ther. 2015 May;23(5):875-84. doi: 10.1038/mt.2015.30. Epub 2015 Feb 20. PMID: 25698151 [PubMed – indexed for MEDLINE].
  31. Gene therapy to rescue retinal degeneration caused by mutations in rhodopsin. Rossmiller BP, Ryals RC, Lewin AS. Methods Mol Biol. 2015;1271:391-410. doi: 10.1007/978-1-4939-2330-4_25. PMID: 25697537 [PubMed – indexed for MEDLINE] Free PMC Article. 
  32. Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium. Thompson DA, Ali RR, Banin E, Branham KE, Flannery JG, Gamm DM, Hauswirth WW, Heckenlively JR, Iannaccone A, Jayasundera KT, Khan NW, Molday RS, Pennesi ME, Reh TA, Weleber RG, Zacks DN; Monaciano Consortium. Invest Ophthalmol Vis Sci. 2015 Feb 9;56(2):918-31. doi: 10.1167/iovs.14-16049. Review. PMID: 25667399 [PubMed – indexed for MEDLINE] Free PMC Article. 
  33. CHIP, a carboxy terminus HSP-70 interacting protein, prevents cell death induced by endoplasmic reticulum stress in the central nervous system. Cabral Miranda F, Adão-Novaes J, Hauswirth WW, Linden R, Petrs-Silva H, Chiarini LB. Front Cell Neurosci. 2015 Jan 9;8:438. doi: 10.3389/fncel.2014.00438. eCollection 2014. PMID: 25620910 [PubMed] Free PMC Article. 
  34. Complex I subunit gene therapy with NDUFA6 ameliorates neurodegeneration in EAE. Talla V, Koilkonda R, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Guy J. Invest Ophthalmol Vis Sci. 2015 Jan 22;56(2):1129-40. doi: 10.1167/iovs.14-15950. PMID: 25613946 [PubMed – indexed for MEDLINE] Free PMC Article. 
  35. Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants. Benskey MJ, Kuhn NC, Galligan JJ, Garcia J, Boye SE, Hauswirth WW, Mueller C, Boye SL, Manfredsson FP. Mol Ther. 2015 Mar;23(3):488-500. doi: 10.1038/mt.2015.7. Epub 2015 Jan 16. PMID: 25592336 [PubMed – indexed for MEDLINE] Free PMC Article. 
  36. Pseudo-fovea formation after gene therapy for RPE65-LCA. Cideciyan AV, Aguirre GK, Jacobson SG, Butt OH, Schwartz SB, Swider M, Roman AJ, Sadigh S, Hauswirth WW. Invest Ophthalmol Vis Sci. 2014 Dec 23;56(1):526-37. doi: 10.1167/iovs.14-15895. PMID: 25537204 [PubMed – indexed for MEDLINE] Free PMC Article. 
  37. STAT3 promotes survival of mutant photoreceptors in inherited photoreceptor degeneration models. Jiang K, Wright KL, Zhu P, Szego MJ, Bramall AN, Hauswirth WW, Li Q, Egan SE, McInnes RR. Proc Natl Acad Sci U S A. 2014 Dec 30;111(52):E5716-23. doi: 10.1073/pnas.1411248112. Epub 2014 Dec 15. PMID: 25512545 [PubMed – indexed for MEDLINE] Free PMC Article. 
  38. AAV-mediated and pharmacological induction of Hsp70 expression stimulates survival of retinal ganglion cells following axonal injury. Kwong JM, Gu L, Nassiri N, Bekerman V, Kumar-Singh R, Rhee KD, Yang XJ, Hauswirth WW, Caprioli J, Piri N. Gene Ther. 2015 Feb;22(2):138-45. doi: 10.1038/gt.2014.105. Epub 2014 Nov 27. PMID: 25427613 [PubMed – indexed for MEDLINE] Free PMC Article. 
  39. Gene delivery of a viral anti-inflammatory protein to combat ocular inflammation. Ildefonso CJ, Jaime H, Rahman MM, Li Q, Boye SE, Hauswirth WW, Lucas AR, McFadden G, Lewin AS. Hum Gene Ther. 2015 Jan;26(1):59-68. doi: 10.1089/hum.2014.089. PMID: 25420215 [PubMed – indexed for MEDLINE] Free PMC Article. 
  40. DICER1/Alu RNA dysmetabolism induces Caspase-8-mediated cell death in age-related macular degeneration. Kim Y, Tarallo V, Kerur N, Yasuma T, Gelfand BD, Bastos-Carvalho A, Hirano Y, Yasuma R, Mizutani T, Fowler BJ, Li S, Kaneko H, Bogdanovich S, Ambati BK, Hinton DR, Hauswirth WW, Hakem R, Wright C, Ambati J. Proc Natl Acad Sci U S A. 2014 Nov 11;111(45):16082-7. doi: 10.1073/pnas.1403814111. Epub 2014 Oct 27. PMID: 25349431 [PubMed – indexed for MEDLINE] Free PMC Article. 
  41. LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile. Koilkonda R, Yu H, Talla V, Porciatti V, Feuer WJ, Hauswirth WW, Chiodo V, Erger KE, Boye SL, Lewin AS, Conlon TJ, Renner L, Neuringer M, Detrisac C, Guy J. Invest Ophthalmol Vis Sci. 2014 Oct 23;55(12):7739-53. doi: 10.1167/iovs.14-15388. PMID: 25342621 [PubMed – indexed for MEDLINE] Free PMC Article. 
  42. Numb regulates the polarized delivery of cyclic nucleotide-gated ion channels in rod photoreceptor cilia. Ramamurthy V, Jolicoeur C, Koutroumbas D, Mühlhans J, Le YZ, Hauswirth WW, Giessl A, Cayouette M. J Neurosci. 2014 Oct 15;34(42):13976-87. doi: 10.1523/JNEUROSCI.1938-14.2014. Erratum in: J Neurosci. 2015 Apr 15;35(15):6254. PMID: 25319694 [PubMed – indexed for MEDLINE] Free PMC Article. 
  43. Gene augmentation for X-linked retinitis pigmentosa caused by mutations in RPGR. Beltran WA, Cideciyan AV, Lewin AS, Hauswirth WW, Jacobson SG, Aguirre GD. Cold Spring Harb Perspect Med. 2014 Oct 9;5(2):a017392. doi: 10.1101/cshperspect.a017392. Review. PMID: 25301933 [PubMed – indexed for MEDLINE].
  44. Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model. Ku CA, Chiodo VA, Boye SL, Hayes A, Goldberg AF, Hauswirth WW, Ramamurthy V. Hum Mol Genet. 2015 Feb 1;24(3):670-84. doi: 10.1093/hmg/ddu487. Epub 2014 Sep 30.  PMID: 25274777 [PubMed – indexed for MEDLINE] Free PMC Article. 
  45. Leber congenital amaurosis caused by mutations in GUCY2D. Boye SE. Cold Spring Harb Perspect Med. 2014 Sep 25;5(1):a017350. doi: 10.1101/cshperspect.a017350. Review. PMID: 25256176 [PubMed – indexed for MEDLINE].
  46. In vivo knockdown of Piccolino disrupts presynaptic ribbon morphology in mouse photoreceptor synapses. Regus-Leidig H, Fuchs M, Löhner M, Leist SR, Leal-Ortiz S, Chiodo VA, Hauswirth WW, Garner CC, Brandstätter JH. Front Cell Neurosci. 2014 Sep 3;8:259. doi: 10.3389/fncel.2014.00259. eCollection 2014. PMID: 25232303 [PubMed] Free PMC Article. 
  47. Flicker cone function in normal and day blind sheep: a large animal model for human achromatopsia caused by CNGA3 mutation. Ezra-Elia R, Banin E, Honig H, Rosov A, Obolensky A, Averbukh E, Hauswirth WW, Gootwine E, Ofri R. Doc Ophthalmol. 2014 Dec;129(3):141-50. doi: 10.1007/s10633-014-9458-6. Epub 2014 Sep 10. PMID: 25204753 [PubMed – indexed for MEDLINE] Free PMC Article. 
  48. Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus. Hauswirth WW. Hum Gene Ther. 2014 Aug;25(8):671-8. doi: 10.1089/hum.2014.2530. No abstract available. PMID: 25136913 [PubMed – indexed for MEDLINE] Free PMC Article.
  49. Gene augmentation for adRP mutations in RHO. Lewin AS, Rossmiller B, Mao H. Cold Spring Harb Perspect Med. 2014 Jul 18;4(9):a017400. doi: 10.1101/cshperspect.a017400. Review. PMID: 25037104 [PubMed – indexed for MEDLINE] Free PMC Article. 
  50. Gene therapy with mitochondrial heat shock protein 70 suppresses visual loss and optic atrophy in experimental autoimmune encephalomyelitis. Talla V, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Guy J. Invest Ophthalmol Vis Sci. 2014 Jul 11;55(8):5214-26. doi: 10.1167/iovs.14-14688. PMID: 25015358 [PubMed – indexed for MEDLINE] Free PMC Article. 
  51. Mitochondrial oxidative stress in the retinal pigment epithelium leads to localized retinal degeneration. Mao H, Seo SJ, Biswal MR, Li H, Conners M, Nandyala A, Jones K, Le YZ, Lewin AS. Invest Ophthalmol Vis Sci. 2014 Jul 1;55(7):4613-27. doi: 10.1167/iovs.14-14633. PMID: 24985474 [PubMed – indexed for MEDLINE] Free PMC Article. 
  52. Genetically targeted binary labeling of retinal neurons. Zhu Y, Xu J, Hauswirth WW, DeVries SH. J Neurosci. 2014 Jun 4;34(23):7845-61. doi: 10.1523/JNEUROSCI.2960-13.2014. PMID: 24899708 [PubMed – indexed for MEDLINE] Free PMC Article. 
  53. Insights gained from gene therapy in animal models of retGC1 deficiency.  Boye SE.  Front Mol Neurosci. 2014 May 14;7:43. doi: 10.3389/fnmol.2014.00043. eCollection 2014. Review. PMID: 24860425 [PubMed] Free PMC Article.
  54. Drug and gene delivery to the back of the eye: from bench to bedside. Rowe-Rendleman CL, Durazo SA, Kompella UB, Rittenhouse KD, Di Polo A, Weiner AL, Grossniklaus HE, Naash MI, Lewin AS, Horsager A, Edelhauser HF. Invest Ophthalmol Vis Sci. 2014 Apr 28;55(4):2714-30. doi: 10.1167/iovs.13-13707. Review. No abstract available. PMID: 24777644 [PubMed – indexed for MEDLINE] Free PMC Article. 
  55. Gene therapy: charting a future course–summary of a National Institutes of Health Workshop, April 12, 2013. O’Reilly M, Federoff HJ, Fong Y, Kohn DB, Patterson AP, Ahmed N, Asokan A, Boye SE, Crystal RG, De Oliveira S, Gargiulo L, Harper SQ, Ikeda Y, Jambou R, Montgomery M, Prograis L, Rosenthal E, Sterman DH, Vandenberghe LH, Zoloth L, Abedi M, Adair J, Adusumilli PS, Goins WF, Gray J, Monahan P, Popplewell L, Sena-Esteves M, Tannous B, Weber T, Wierda W, Gopal-Srivastava R, McDonald CL, Rosenblum D, Corrigan-Curay J. Hum Gene Ther. 2014 Jun;25(6):488-97. doi: 10.1089/hum.2014.045. Review.PMID: 24773122 [PubMed – indexed for MEDLINE] Free PMC Article. 
  56. Targeted CNS Delivery Using Human MiniPromoters and Demonstrated Compatibility with Adeno-Associated Viral Vectors. de Leeuw CN, Dyka FM, Boye SL, Laprise S, Zhou M, Chou AY, Borretta L, McInerny SC, Banks KG, Portales-Casamar E, Swanson MI, D’Souza CA, Boye SE, Jones SJ, Holt RA, Goldowitz D, Hauswirth WW, Wasserman WW, Simpson EM. Mol Ther Methods Clin Dev. 2014 Jan 8;1:5.  PMID: 24761428 [PubMed] Free PMC Article. 
  57. Inhibitor of apoptosis-stimulating protein of p53 (iASPP) is required for neuronal survival after axonal injury. Wilson AM, Chiodo VA, Boye SL, Brecha NC, Hauswirth WW, Di Polo A. PLoS One. 2014 Apr 8;9(4):e94175. doi: 10.1371/journal.pone.0094175. eCollection 2014. PMID: 24714389 [PubMed – indexed for MEDLINE] Free PMC Article. 
  58. Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy. Boye SE, Huang WC, Roman AJ, Sumaroka A, Boye SL, Ryals RC, Olivares MB, Ruan Q, Tucker BA, Stone EM, Swaroop A, Cideciyan AV, Hauswirth WW, Jacobson SG.  PLoS One. 2014 Mar 26;9(3):e92928. doi: 10.1371/journal.pone.0092928. eCollection 2014. PMID: 24671090 [PubMed – indexed for MEDLINE] Free PMC Article. 
  59. Gene therapy in the rd6 mouse model of retinal degeneration. Dinculescu A, Min SH, Deng WT, Li Q, Hauswirth WW. Adv Exp Med Biol. 2014;801:711-8. doi: 10.1007/978-1-4614-3209-8_89. PMID: 24664762 [PubMed – indexed for MEDLINE].
  60. Cone specific promoter for use in gene therapy of retinal degenerative diseases. Dyka FM, Boye SL, Ryals RC, Chiodo VA, Boye SE, Hauswirth WW. Adv Exp Med Biol. 2014;801:695-701. doi: 10.1007/978-1-4614-3209-8_87. PMID: 24664760 [PubMed – indexed for MEDLINE] Free PMC Article.  
  61. Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A. Dyka FM, Boye SL, Chiodo VA, Hauswirth WW, Boye SE. Hum Gene Ther Methods. 2014 Apr;25(2):166-77. doi: 10.1089/hgtb.2013.212. PMID: 24568220 [PubMed – indexed for MEDLINE] Free PMC Article. 
  62. AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice. Dai X, Han J, Qi Y, Zhang H, Xiang L, Lv J, Li J, Deng WT, Chang B, Hauswirth WW, Pang JJ. Invest Ophthalmol Vis Sci. 2014 Mar 20;55(3):1724-34. doi: 10.1167/iovs.13-13654. PMID: 24557352 [PubMed – indexed for MEDLINE] Free PMC Article. 
  63. Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial. Koilkonda RD, Yu H, Chou TH, Feuer WJ, Ruggeri M, Porciatti V, Tse D, Hauswirth WW, Chiodo V, Boye SL, Lewin AS, Neuringer M, Renner L, Guy J. JAMA Ophthalmol. 2014 Apr 1;132(4):409-20. doi: 10.1001/jamaophthalmol.2013.7630. PMID: 24457989 [PubMed – indexed for MEDLINE] Free PMC Article. 
  64. Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach. Mowat FM, Gornik KR, Dinculescu A, Boye SL, Hauswirth WW, Petersen-Jones SM, Bartoe JT. Gene Ther. 2014 Jan;21(1):96-105. doi: 10.1038/gt.2013.64. Epub 2013 Nov 14. PMID: 24225638 [PubMed – indexed for MEDLINE] Free PMC Article.
  65. Reprogramming adipose tissue-derived mesenchymal stem cells into pluripotent stem cells by a mutant adeno-associated viral vector. Chen MJ, Lu Y, Hamazaki T, Tsai HY, Erger K, Conlon T, Elshikha AS, Li H, Srivastava A, Yao C, Brantly M, Chiodo V, Hauswirth W, Terada N, Song S. Hum Gene Ther Methods. 2014 Feb;25(1):72-82. doi: 10.1089/hgtb.2013.011. Epub 2013 Dec 28. PMID: 24191859 [PubMed – indexed for MEDLINE] Free PMC Article. 
  66. Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects. Guziewicz KE, Zangerl B, Komáromy AM, Iwabe S, Chiodo VA, Boye SL, Hauswirth WW, Beltran WA, Aguirre GD. PLoS One. 2013 Oct 15;8(10):e75666. doi: 10.1371/journal.pone.0075666. eCollection 2013. PMID: 24143172 [PubMed – indexed for MEDLINE] Free PMC Article. 
  67. Next-generation sequencing of mitochondrial targeted AAV transfer of human ND4 in mice. Yu H, Mehta A, Wang G, Hauswirth WW, Chiodo V, Boye SL, Guy J. Mol Vis. 2013 Jul 14;19:1482-91. Print 2013. PMID: 23869167 [PubMed – indexed for MEDLINE] Free PMC Article. 
  68. Cone phosphodiesterase-6α’ restores rod function and confers distinct physiological properties in the rod phosphodiesterase-6β-deficient rd10 mouse. Deng WT, Sakurai K, Kolandaivelu S, Kolesnikov AV, Dinculescu A, Li J, Zhu P, Liu X, Pang J, Chiodo VA, Boye SL, Chang B, Ramamurthy V, Kefalov VJ, Hauswirth WW. J Neurosci. 2013 Jul 17;33(29):11745-53. doi: 10.1523/JNEUROSCI.1536-13.2013. PMID: 23864662 [PubMed – indexed for MEDLINE] Free PMC Article.
  69. Photoreceptor avascular privilege is shielded by soluble VEGF receptor-1. Luo L, Uehara H, Zhang X, Das SK, Olsen T, Holt D, Simonis JM, Jackman K, Singh N, Miya TR, Huang W, Ahmed F, Bastos-Carvalho A, Le YZ, Mamalis C, Chiodo VA, Hauswirth WW, Baffi J, Lacal PM, Orecchia A, Ferrara N, Gao G, Young-Hee K, Fu Y, Owen L, Albuquerque R, Baehr W, Thomas K, Li DY, Chalam KV, Shibuya M, Grisanti S, Wilson DJ, Ambati J, Ambati BK. Elife. 2013 Jun 18;2:e00324. doi: 10.7554/eLife.00324. PMID: 23795287 [PubMed – indexed for MEDLINE] Free PMC Article. 
  70. NADH-dehydrogenase type-2 suppresses irreversible visual loss and neurodegeneration in the EAE animal model of MS. Talla V, Yu H, Chou TH, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Lewin AS, Guy J. Mol Ther. 2013 Oct;21(10):1876-88. doi: 10.1038/mt.2013.104. Epub 2013 Jun 11. PMID: 23752309 [PubMed – indexed for MEDLINE] Free PMC Article. 
  71. RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12. Molday LL, Djajadi H, Yan P, Szczygiel L, Boye SL, Chiodo VA, Gregory-Evans K, Sarunic MV, Hauswirth WW, Molday RS. Hum Mol Genet. 2013 Oct 1;22(19):3894-905. doi: 10.1093/hmg/ddt244. Epub 2013 Jun 4. PMID: 23740938 [PubMed – indexed for MEDLINE] Free PMC Article. 
  72. Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa. Conlon TJ, Deng WT, Erger K, Cossette T, Pang JJ, Ryals R, Clément N, Cleaver B, McDoom I, Boye SE, Peden MC, Sherwood MB, Abernathy CR, Alkuraya FS, Boye SL, Hauswirth WW. Hum Gene Ther Clin Dev. 2013 Mar;24(1):23-8. doi: 10.1089/humc.2013.037. Epub 2013 Apr 3. PMID: 23692380 [PubMed – indexed for MEDLINE] Free PMC Article. 
  73. Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors. Kay CN, Ryals RC, Aslanidi GV, Min SH, Ruan Q, Sun J, Dyka FM, Kasuga D, Ayala AE, Van Vliet K, Agbandje-McKenna M, Hauswirth WW, Boye SL, Boye SE. PLoS One. 2013 Apr 26;8(4):e62097. doi: 10.1371/journal.pone.0062097. Print 2013. Erratum in: PLoS One. 2013;8(9). doi:10.1371/annotation/99ee1789-a658-4fb0-8593-40a40e9f344a. PMID: 23637972 [PubMed – indexed for MEDLINE] Free PMC Article. 
  74. Transient photoreceptor deconstruction by CNTF enhances rAAV-mediated cone functional rescue in late stage CNGB3-achromatopsia. Komáromy AM, Rowlan JS, Corr AT, Reinstein SL, Boye SL, Cooper AE, Gonzalez A, Levy B, Wen R, Hauswirth WW, Beltran WA, Aguirre GD. Mol Ther. 2013 Jun;21(6):1131-41. doi: 10.1038/mt.2013.50. Epub 2013 Apr 9. PMID: 23568263 [PubMed – indexed for MEDLINE] Free PMC Article. 
  75. Evaluation of lateral spread of transgene expression following subretinal AAV-mediated gene delivery in dogs. Bruewer AR, Mowat FM, Bartoe JT, Boye SL, Hauswirth WW, Petersen-Jones SM. PLoS One. 2013;8(4):e60218. doi: 10.1371/journal.pone.0060218. Epub 2013 Apr 3. PMID: 23560080 [PubMed – indexed for MEDLINE] Free PMC Article. 
  76. RNAi-mediated gene suppression in a GCAP1(L151F) cone-rod dystrophy mouse model. Jiang L, Li TZ, Boye SE, Hauswirth WW, Frederick JM, Baehr W. PLoS One. 2013;8(3):e57676. doi: 10.1371/journal.pone.0057676. Epub 2013 Mar 5. PMID: 23472098 [PubMed – indexed for MEDLINE] Free PMC Article. 
  77. Endothelin-2-mediated protection of mutant photoreceptors in inherited photoreceptor degeneration. Bramall AN, Szego MJ, Pacione LR, Chang I, Diez E, D’Orleans-Juste P, Stewart DJ, Hauswirth WW, Yanagisawa M, McInnes RR. PLoS One. 2013;8(2):e58023. doi: 10.1371/journal.pone.0058023. Epub 2013 Feb 28. PMID: 23469133 [PubMed – indexed for MEDLINE] Free PMC Article. 
  78. Functional genomic screening identifies dual leucine zipper kinase as a key mediator of retinal ganglion cell death. Welsbie DS, Yang Z, Ge Y, Mitchell KL, Zhou X, Martin SE, Berlinicke CA, Hackler L Jr, Fuller J, Fu J, Cao LH, Han B, Auld D, Xue T, Hirai S, Germain L, Simard-Bisson C, Blouin R, Nguyen JV, Davis CH, Enke RA, Boye SL, Merbs SL, Marsh-Armstrong N, Hauswirth WW, DiAntonio A, Nickells RW, Inglese J, Hanes J, Yau KW, Quigley HA, Zack DJ. Proc Natl Acad Sci U S A. 2013 Mar 5;110(10):4045-50. doi: 10.1073/pnas.1211284110. Epub 2013 Feb 19. PMID: 23431148 [PubMed – indexed for MEDLINE] Free PMC Article. 
  79. A comprehensive review of retinal gene therapy. Boye SE, Boye SL, Lewin AS, Hauswirth WW. Mol Ther. 2013 Mar;21(3):509-19. doi: 10.1038/mt.2012.280. Epub 2013 Jan 29. Review. Erratum in: Mol Ther. 2013 Nov;21(11):2130. PMID: 23358189 [PubMed – indexed for MEDLINE] Free PMC Article. 
  80. Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. Lopes VS, Boye SE, Louie CM, Boye S, Dyka F, Chiodo V, Fofo H, Hauswirth WW, Williams DS. Gene Ther. 2013 Aug;20(8):824-33. doi: 10.1038/gt.2013.3. Epub 2013 Jan 24. PMID: 23344065 [PubMed – indexed for MEDLINE] Free PMC Article. 
  81. Imaging the response of the retina to electrical stimulation with genetically encoded calcium indicators. Weitz AC, Behrend MR, Lee NS, Klein RL, Chiodo VA, Hauswirth WW, Humayun MS, Weiland JD, Chow RH. J Neurophysiol. 2013 Apr;109(7):1979-88. doi: 10.1152/jn.00852.2012. Epub 2013 Jan 23. PMID: 23343890 [PubMed – indexed for MEDLINE] Free PMC Article. 
  82. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Cideciyan AV, Jacobson SG, Beltran WA, Sumaroka A, Swider M, Iwabe S, Roman AJ, Olivares MB, Schwartz SB, Komáromy AM, Hauswirth WW, Aguirre GD. Proc Natl Acad Sci U S A. 2013 Feb 5;110(6):E517-25. doi: 10.1073/pnas.1218933110. Epub 2013 Jan 22. PMID: 23341635 [PubMed – indexed for MEDLINE] Free PMC Article. 
  83. Comparison of the transduction efficiency of tyrosine-mutant adeno-associated virus serotype vectors in kidney. Qi YF, Li QH, Shenoy V, Zingler M, Jun JY, Verma A, Katovich MJ, Raizada MK. Clin Exp Pharmacol Physiol. 2013 Jan;40(1):53-5. doi: 10.1111/1440-1681.12037. PMID: 23216315 [PubMed – indexed for MEDLINE] Free PMC Article. 
  84. AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis. Boye SL, Peshenko IV, Huang WC, Min SH, McDoom I, Kay CN, Liu X, Dyka FM, Foster TC, Umino Y, Karan S, Jacobson SG, Baehr W, Dizhoor A, Hauswirth WW, Boye SE. Hum Gene Ther. 2013 Feb;24(2):189-202. doi: 10.1089/hum.2012.193. PMID: 23210611 [PubMed – indexed for MEDLINE] Free PMC Article. 
  85. Development of an anti-angiogenic therapeutic model combining scAAV2-delivered siRNAs and noninvasive photoacoustic imaging of tumor vasculature development. Ruan Q, Xi L, Boye SL, Han S, Chen ZJ, Hauswirth WW, Lewin AS, Boulton ME, Law BK, Jiang WG, Jiang H, Cai J. Cancer Lett. 2013 May 10;332(1):120-9. doi: 10.1016/j.canlet.2012.11.016. Epub 2012 Nov 27. PMID: 23196055 [PubMed – indexed for MEDLINE] Free PMC Article.  
  86. scAAV-mediated gene transfer of interleukin-1-receptor antagonist to synovium and articular cartilage in large mammalian joints. Watson RS, Broome TA, Levings PP, Rice BL, Kay JD, Smith AD, Gouze E, Gouze JN, Dacanay EA, Hauswirth WW, Nickerson DM, Dark MJ, Colahan PT, Ghivizzani SC. Gene Ther. 2013 Jun;20(6):670-7. doi: 10.1038/gt.2012.81. Epub 2012 Nov 15. PMID: 23151520 [PubMed – indexed for MEDLINE] Free PMC Article.  
  87. Gene therapy in animal models of autosomal dominant retinitis pigmentosa. Rossmiller B, Mao H, Lewin AS. Mol Vis. 2012;18:2479-96. Epub 2012 Oct 6. Review. PMID: 23077406 [PubMed – indexed for MEDLINE] Free PMC Article.  
  88. ERK1/2 activation is a therapeutic target in age-related macular degeneration. Dridi S, Hirano Y, Tarallo V, Kim Y, Fowler BJ, Ambati BK, Bogdanovich S, Chiodo VA, Hauswirth WW, Kugel JF, Goodrich JA, Ponicsan SL, Hinton DR, Kleinman ME, Baffi JZ, Gelfand BD, Ambati J. Proc Natl Acad Sci U S A. 2012 Aug 21;109(34):13781-6. doi: 10.1073/pnas.1206494109. Epub 2012 Aug 6. PMID: 22869729 [PubMed – indexed for MEDLINE] Free PMC Article.
  89. The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina. Boye SE, Alexander JJ, Boye SL, Witherspoon CD, Sandefer KJ, Conlon TJ, Erger K, Sun J, Ryals R, Chiodo VA, Clark ME, Girkin CA, Hauswirth WW, Gamlin PD. Hum Gene Ther. 2012 Oct;23(10):1101-15. doi: 10.1089/hum.2012.125. Epub 2012 Sep 20. PMID: 22845794 [PubMed – indexed for MEDLINE] Free PMC Article. 
  90. Organizational motifs for ground squirrel cone bipolar cells. Light AC, Zhu Y, Shi J, Saszik S, Lindstrom S, Davidson L, Li X, Chiodo VA, Hauswirth WW, Li W, DeVries SH. J Comp Neurol. 2012 Sep 1;520(13):2864-87. doi: 10.1002/cne.23068. PMID: 22778006 [PubMed – indexed for MEDLINE] Free PMC Article. 
  91. Mutant NADH dehydrogenase subunit 4 gene delivery to mitochondria by targeting sequence-modified adeno-associated virus induces visual loss and optic atrophy in mice. Yu H, Ozdemir SS, Koilkonda RD, Chou TH, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Lewin AS, Guy J. Mol Vis. 2012;18:1668-83. Epub 2012 Jun 20. PMID: 22773905 [PubMed – indexed for MEDLINE] Free PMC Article. 
  92. Pathological consequences of long-term mitochondrial oxidative stress in the mouse retinal pigment epithelium. Seo SJ, Krebs MP, Mao H, Jones K, Conners M, Lewin AS. Exp Eye Res. 2012 Aug;101:60-71. doi: 10.1016/j.exer.2012.05.013. Epub 2012 Jun 8. PMID: 22687918 [PubMed – indexed for MEDLINE] Free PMC Article. 
  93. Caspase inhibition with XIAP as an adjunct to AAV vector gene-replacement therapy: improving efficacy and prolonging the treatment window. Yao J, Jia L, Khan N, Zheng QD, Moncrief A, Hauswirth WW, Thompson DA, Zacks DN. PLoS One. 2012;7(5):e37197. doi: 10.1371/journal.pone.0037197. Epub 2012 May 16. PMID: 22615940 [PubMed – indexed for MEDLINE] Free PMC Article. 
  94. ER stress is involved in T17M rhodopsin-induced retinal degeneration. Kunte MM, Choudhury S, Manheim JF, Shinde VM, Miura M, Chiodo VA, Hauswirth WW, Gorbatyuk OS, Gorbatyuk MS. Invest Ophthalmol Vis Sci. 2012 Jun 20;53(7):3792-800. doi: 10.1167/iovs.11-9235. PMID: 22589437 [PubMed – indexed for MEDLINE] Free PMC Article.  
  95. DICER1 loss and Alu RNA induce age-related macular degeneration via the NLRP3 inflammasome and MyD88. Tarallo V, Hirano Y, Gelfand BD, Dridi S, Kerur N, Kim Y, Cho WG, Kaneko H, Fowler BJ, Bogdanovich S, Albuquerque RJ, Hauswirth WW, Chiodo VA, Kugel JF, Goodrich JA, Ponicsan SL, Chaudhuri G, Murphy MP, Dunaief JL, Ambati BK, Ogura Y, Yoo JW, Lee DK, Provost P, Hinton DR, Núñez G, Baffi JZ, Kleinman ME, Ambati J. Cell. 2012 May 11;149(4):847-59. doi: 10.1016/j.cell.2012.03.036. Epub 2012 Apr 26. PMID: 22541070 [PubMed – indexed for MEDLINE] Free PMC Article. 
  96. Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber’s hereditary optic neuropathy in a mouse model. Yu H, Koilkonda RD, Chou TH, Porciatti V, Ozdemir SS, Chiodo V, Boye SL, Boye SE, Hauswirth WW, Lewin AS, Guy J. Proc Natl Acad Sci U S A. 2012 May 15;109(20):E1238-47. doi: 10.1073/pnas.1119577109. Epub 2012 Apr 20. PMID: 22523243 [PubMed – indexed for MEDLINE] Free PMC Article. 
  97. AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia. Pang JJ, Deng WT, Dai X, Lei B, Everhart D, Umino Y, Li J, Zhang K, Mao S, Boye SL, Liu L, Chiodo VA, Liu X, Shi W, Tao Y, Chang B, Hauswirth WW. PLoS One. 2012;7(4):e35250. doi: 10.1371/journal.pone.0035250. Epub 2012 Apr 11. Erratum in: PLoS One. 2014;9(1). doi:10.1371/annotation/29fb0ebc-b1c7-4d05-bd70-d29b10299df4. PMID: 22509403 [PubMed – indexed for MEDLINE] Free PMC Article. 
  98. The 5HT1a receptor agonist 8-Oh DPAT induces protection from lipofuscin accumulation and oxidative stress in the retinal pigment epithelium. Thampi P, Rao HV, Mitter SK, Cai J, Mao H, Li H, Seo S, Qi X, Lewin AS, Romano C, Boulton ME. PLoS One. 2012;7(4):e34468. doi: 10.1371/journal.pone.0034468. Epub 2012 Apr 3. PMID: 22509307 [PubMed – indexed for MEDLINE] Free PMC Article.
  99. Glucose regulated protein 78 diminishes α-synuclein neurotoxicity in a rat model of Parkinson disease. Gorbatyuk MS, Shabashvili A, Chen W, Meyers C, Sullivan LF, Salganik M, Lin JH, Lewin AS, Muzyczka N, Gorbatyuk OS. Mol Ther. 2012 Jul;20(7):1327-37. doi: 10.1038/mt.2012.28. Epub 2012 Mar 20. PMID: 22434142 [PubMed – indexed for MEDLINE] Free PMC Article. 
  100. Tyrosine-mutant AAV8 delivery of human MERTK provides long-term retinal preservation in RCS rats. Deng WT, Dinculescu A, Li Q, Boye SL, Li J, Gorbatyuk MS, Pang J, Chiodo VA, Matthes MT, Yasumura D, Liu L, Alkuraya FS, Zhang K, Vollrath D, LaVail MM, Hauswirth WW. Invest Ophthalmol Vis Sci. 2012 Apr 6;53(4):1895-904. doi: 10.1167/iovs.11-8831. PMID: 22408006 [PubMed – indexed for MEDLINE] Free PMC Article.
  101. Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. Beltran WA, Cideciyan AV, Lewin AS, Iwabe S, Khanna H, Sumaroka A, Chiodo VA, Fajardo DS, Román AJ, Deng WT, Swider M, Alemán TS, Boye SL, Genini S, Swaroop A, Hauswirth WW, Jacobson SG, Aguirre GD. Proc Natl Acad Sci U S A. 2012 Feb 7;109(6):2132-7. doi: 10.1073/pnas.1118847109. Epub 2012 Jan 23. PMID: 22308428 [PubMed – indexed for MEDLINE] Free PMC Article. 
  102. Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice. Mao H, Gorbatyuk MS, Rossmiller B, Hauswirth WW, Lewin AS.  Hum Gene Ther. 2012 Apr;23(4):356-66. doi: 10.1089/hum.2011.213. Epub 2012 Mar 28. PMID: 22289036 [PubMed – indexed for MEDLINE] Free PMC Article. 
  103. Towards optogenetic sensory replacement. Doroudchi MM, Greenberg KP, Zorzos AN, Hauswirth WW, Fonstad CG, Horsager A, Boyden ES. Conf Proc IEEE Eng Med Biol Soc. 2011;2011:3139-41. doi: 10.1109/IEMBS.2011.6090856. PMID: 22255005 [PubMed – indexed for MEDLINE] Free PMC Article. 
  104. γ-Secretase inhibition of murine choroidal neovascularization is associated with reduction of superoxide and proinflammatory cytokines. Qi X, Cai J, Ruan Q, Liu L, Boye SL, Chen Z, Hauswirth WW, Ryals RC, Shaw L, Caballero S, Grant MB, Boulton ME. Invest Ophthalmol Vis Sci. 2012 Feb 1;53(2):574-85. doi: 10.1167/iovs.11-8728. Print 2012 Feb. PMID: 22205609 [PubMed – indexed for MEDLINE] Free PMC Article. 
  105. Suppression of rds expression by siRNA and gene replacement strategies for gene therapy using rAAV vector. Petrs-Silva H, Yasumura D, Matthes MT, LaVail MM, Lewin AS, Hauswirth WW. Adv Exp Med Biol. 2012;723:215-23. doi: 10.1007/978-1-4614-0631-0_29. No abstract available. PMID: 22183336 [PubMed – indexed for MEDLINE] Free PMC Article. 
  106. Gene delivery of wild-type rhodopsin rescues retinal function in an autosomal dominant retinitis pigmentosa mouse model. Mao H, Gorbatyuk MS, Hauswirth WW, Lewin AS. Adv Exp Med Biol. 2012;723:199-205. doi: 10.1007/978-1-4614-0631-0_27. No abstract available. PMID: 22183334 [PubMed – indexed for MEDLINE] Free PMC Article. 
  107. Functional rescue of P23H rhodopsin photoreceptors by gene delivery. Gorbatyuk MS, Gorbatyuk OS, LaVail MM, Lin JH, Hauswirth WW, Lewin AS. Adv Exp Med Biol. 2012;723:191-7. doi: 10.1007/978-1-4614-0631-0_26. No abstract available. PMID: 22183333 [PubMed – indexed for MEDLINE] Free PMC Article.
  108. Gene therapy for retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of concept. Dinculescu A, Estreicher J, Zenteno JC, Aleman TS, Schwartz SB, Huang WC, Roman AJ, Sumaroka A, Li Q, Deng WT, Min SH, Chiodo VA, Neeley A, Liu X, Shu X, Matias-Florentino M, Buentello-Volante B, Boye SL, Cideciyan AV, Hauswirth WW, Jacobson SG. Hum Gene Ther. 2012 Apr;23(4):367-76. doi: 10.1089/hum.2011.169. Epub 2012 Jan 26. PMID: 22142163 [PubMed – indexed for MEDLINE] Free PMC Article. 
  109. Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model. Jiang L, Zhang H, Dizhoor AM, Boye SE, Hauswirth WW, Frederick JM, Baehr W. Proc Natl Acad Sci U S A. 2011 Nov 8;108(45):18476-81. doi: 10.1073/pnas.1112758108. Epub 2011 Oct 31. PMID: 22042849 [PubMed – indexed for MEDLINE] Free PMC Article. 
  110. γ-Secretase and presenilin mediate cleavage and phosphorylation of vascular endothelial growth factor receptor-1.  Cai J, Chen Z, Ruan Q, Han S, Liu L, Qi X, Boye SL, Hauswirth WW, Grant MB, Boulton ME. J Biol Chem. 2011 Dec 9;286(49):42514-23. doi: 10.1074/jbc.M111.296590. Epub 2011 Oct 20.  PMID: 22016384 [PubMed – indexed for MEDLINE] Free PMC Article.
  111. Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Jacobson SG, Cideciyan AV, Ratnakaram R, Heon E, Schwartz SB, Roman AJ, Peden MC, Aleman TS, Boye SL, Sumaroka A, Conlon TJ, Calcedo R, Pang JJ, Erger KE, Olivares MB, Mullins CL, Swider M, Kaushal S, Feuer WJ, Iannaccone A, Fishman GA, Stone EM, Byrne BJ, Hauswirth WW. Arch Ophthalmol. 2012 Jan;130(1):9-24. doi: 10.1001/archophthalmol.2011.298. Epub 2011 Sep 12. PMID: 21911650 [PubMed – indexed for MEDLINE] Free PMC Article.
  112. Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis. Ku CA, Chiodo VA, Boye SL, Goldberg AF, Li T, Hauswirth WW, Ramamurthy V. Hum Mol Genet. 2011 Dec 1;20(23):4569-81. doi: 10.1093/hmg/ddr391. Epub 2011 Aug 31. PMID: 21880665 [PubMed – indexed for MEDLINE] Free PMC Article.
  113. ACE2 and Ang-(1-7) confer protection against development of diabetic retinopathy. Verma A, Shan Z, Lei B, Yuan L, Liu X, Nakagawa T, Grant MB, Lewin AS, Hauswirth WW, Raizada MK, Li Q. Mol Ther. 2012 Jan;20(1):28-36. doi: 10.1038/mt.2011.155. Epub 2011 Jul 26. PMID: 21792177 [PubMed – indexed for MEDLINE] Free PMC Article. 
  114. Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse. Boye SL, Conlon T, Erger K, Ryals R, Neeley A, Cossette T, Pang J, Dyka FM, Hauswirth WW, Boye SE. Invest Ophthalmol Vis Sci. 2011 Sep 9;52(10):7098-108. doi: 10.1167/iovs.11-7867. PMID: 21778276 [PubMed – indexed for MEDLINE] Free PMC Article. 
  115. Efficient cochlear gene transfection in guinea-pigs with adeno-associated viral vectors by partial digestion of round window membrane. Wang H, Murphy R, Taaffe D, Yin S, Xia L, Hauswirth WW, Bance M, Robertson GS, Wang J. Gene Ther. 2012 Mar;19(3):255-63. doi: 10.1038/gt.2011.91. Epub 2011 Jun 23. PMID: 21697953 [PubMed – indexed for MEDLINE] Free PMC Article.
  116. PEDF regulates vascular permeability by a γ-secretase-mediated pathway. Cai J, Wu L, Qi X, Li Calzi S, Caballero S, Shaw L, Ruan Q, Grant MB, Boulton ME. PLoS One. 2011;6(6):e21164. doi: 10.1371/journal.pone.0021164. Epub 2011 Jun 17. PMID: 21695048 [PubMed – indexed for MEDLINE] Free PMC Article. 
  117. Quantifying transduction efficiencies of unmodified and tyrosine capsid mutant AAV vectors in vitro using two ocular cell lines. Ryals RC, Boye SL, Dinculescu A, Hauswirth WW, Boye SE. Mol Vis. 2011 Apr 29;17:1090-102. PMID: 21552473 [PubMed – indexed for MEDLINE] Free PMC Article.  
  118. Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness. Doroudchi MM, Greenberg KP, Liu J, Silka KA, Boyden ES, Lockridge JA, Arman AC, Janani R, Boye SE, Boye SL, Gordon GM, Matteo BC, Sampath AP, Hauswirth WW, Horsager A. Mol Ther. 2011 Jul;19(7):1220-9. doi: 10.1038/mt.2011.69. Epub 2011 Apr 19. PMID: 21505421 [PubMed – indexed for MEDLINE] Free PMC Article. 
  119. Efficient mutagenesis of the rhodopsin gene in rod photoreceptor neurons in mice. Chan F, Hauswirth WW, Wensel TG, Wilson JH. Nucleic Acids Res. 2011 Aug;39(14):5955-66. doi: 10.1093/nar/gkr196. Epub 2011 Apr 7. PMID: 21478169 [PubMed – indexed for MEDLINE] Free PMC Article. 
  120. Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model. Simons DL, Boye SL, Hauswirth WW, Wu SM. Proc Natl Acad Sci U S A. 2011 Apr 12;108(15):6276-81. doi: 10.1073/pnas.1019222108. Epub 2011 Mar 28. PMID: 21444805 [PubMed – indexed for MEDLINE] Free PMC Article. 
  121. AAV5-mediated sFLT01 gene therapy arrests retinal lesions in Ccl2(-/-)/Cx3cr1(-/-) mice. Tuo J, Pang JJ, Cao X, Shen D, Zhang J, Scaria A, Wadsworth SC, Pechan P, Boye SL, Hauswirth WW, Chan CC. Neurobiol Aging. 2012 Feb;33(2):433.e1-10. doi: 10.1016/j.neurobiolaging.2011.01.009. Epub 2011 Mar 12. PMID: 21397984 [PubMed – indexed for MEDLINE] Free PMC Article.  
  122. Ab-externo AAV-mediated gene delivery to the suprachoroidal space using a 250 micron flexible microcatheter. Peden MC, Min J, Meyers C, Lukowski Z, Li Q, Boye SL, Levine M, Hauswirth WW, Ratnakaram R, Dawson W, Smith WC, Sherwood MB. PLoS One. 2011 Feb 11;6(2):e17140. doi: 10.1371/journal.pone.0017140. PMID: 21347253 [PubMed – indexed for MEDLINE] Free PMC Article. 
  123. DICER1 deficit induces Alu RNA toxicity in age-related macular degeneration. Kaneko H, Dridi S, Tarallo V, Gelfand BD, Fowler BJ, Cho WG, Kleinman ME, Ponicsan SL, Hauswirth WW, Chiodo VA, Karikó K, Yoo JW, Lee DK, Hadziahmetovic M, Song Y, Misra S, Chaudhuri G, Buaas FW, Braun RE, Hinton DR, Zhang Q, Grossniklaus HE, Provis JM, Madigan MC, Milam AH, Justice NL, Albuquerque RJ, Blandford AD, Bogdanovich S, Hirano Y, Witta J, Fuchs E, Littman DR, Ambati BK, Rudin CM, Chong MM, Provost P, Kugel JF, Goodrich JA, Dunaief JL, Baffi JZ, Ambati J. Nature. 2011 Mar 17;471(7338):325-30. doi: 10.1038/nature09830. Epub 2011 Feb 6. PMID: 21297615 [PubMed – indexed for MEDLINE] Free PMC Article. 
  124. Whirlin replacement restores the formation of the USH2 protein complex in whirlin knockout photoreceptors. Zou J, Luo L, Shen Z, Chiodo VA, Ambati BK, Hauswirth WW, Yang J. Invest Ophthalmol Vis Sci. 2011 Apr 12;52(5):2343-51. doi: 10.1167/iovs.10-6141. Print 2011 Apr. PMID: 21212183 [PubMed – indexed for MEDLINE] Free PMC Article. 
  125. Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis. Li X, Li W, Dai X, Kong F, Zheng Q, Zhou X, Lü F, Chang B, Rohrer B, Hauswirth WW, Qu J, Pang JJ. Invest Ophthalmol Vis Sci. 2011 Jan 5;52(1):7-15. doi: 10.1167/iovs.10-6138. Print 2011 Jan. PMID: 21169527 [PubMed – indexed for MEDLINE] Free PMC Article. 
  126. Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa. Pang JJ, Dai X, Boye SE, Barone I, Boye SL, Mao S, Everhart D, Dinculescu A, Liu L, Umino Y, Lei B, Chang B, Barlow R, Strettoi E, Hauswirth WW. Mol Ther. 2011 Feb;19(2):234-42. doi: 10.1038/mt.2010.273. Epub 2010 Dec 7. PMID: 21139570 [PubMed – indexed for MEDLINE] Free PMC Article. 
  127. AAV delivery of wild-type rhodopsin preserves retinal function in a mouse model of autosomal dominant retinitis pigmentosa. Mao H, James T Jr, Schwein A, Shabashvili AE, Hauswirth WW, Gorbatyuk MS, Lewin AS. Hum Gene Ther. 2011 May;22(5):567-75. doi: 10.1089/hum.2010.140. Epub 2011 Mar 7. PMID: 21126223 [PubMed – indexed for MEDLINE] Free PMC Article. 
  128. Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina. Petrs-Silva H, Dinculescu A, Li Q, Deng WT, Pang JJ, Min SH, Chiodo V, Neeley AW, Govindasamy L, Bennett A, Agbandje-McKenna M, Zhong L, Li B, Jayandharan GR, Srivastava A, Lewin AS, Hauswirth WW. Mol Ther. 2011 Feb;19(2):293-301. doi: 10.1038/mt.2010.234. Epub 2010 Nov 2. PMID: 21045809 [PubMed – indexed for MEDLINE] Free PMC Article. 
  129. Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule. Lukason M, DuFresne E, Rubin H, Pechan P, Li Q, Kim I, Kiss S, Flaxel C, Collins M, Miller J, Hauswirth W, Maclachlan T, Wadsworth S, Scaria A. Mol Ther. 2011 Feb;19(2):260-5. doi: 10.1038/mt.2010.230. Epub 2010 Oct 26. PMID: 20978476 [PubMed – indexed for MEDLINE] Free PMC Article. 
  130. XIAP therapy increases survival of transplanted rod precursors in a degenerating host retina. Yao J, Feathers KL, Khanna H, Thompson D, Tsilfidis C, Hauswirth WW, Heckenlively JR, Swaroop A, Zacks DN. Invest Ophthalmol Vis Sci. 2011 Mar 1;52(3):1567-72. doi: 10.1167/iovs.10-5998. Print 2011 Mar. PMID: 20926819 [PubMed – indexed for MEDLINE] Free PMC Article. 
  131. Leber hereditary optic neuropathy gene therapy clinical trial recruitment: year 1. Lam BL, Feuer WJ, Abukhalil F, Porciatti V, Hauswirth WW, Guy J. Arch Ophthalmol. 2010 Sep;128(9):1129-35. doi: 10.1001/archophthalmol.2010.201. PMID: 20837795 [PubMed – indexed for MEDLINE] Free PMC Article.
  132. Induction of rapid and highly efficient expression of the human ND4 complex I subunit in the mouse visual system by self-complementary adeno-associated virus. Koilkonda RD, Chou TH, Porciatti V, Hauswirth WW, Guy J. Arch Ophthalmol. 2010 Jul;128(7):876-83. doi: 10.1001/archophthalmol.2010.135. PMID: 20625049 [PubMed – indexed for MEDLINE] Free PMC Article.
  133. Molecular anthropology meets genetic medicine to treat blindness in the North African Jewish population: human gene therapy initiated in Israel. Banin E, Bandah-Rozenfeld D, Obolensky A, Cideciyan AV, Aleman TS, Marks-Ohana D, Sela M, Boye S, Sumaroka A, Roman AJ, Schwartz SB, Hauswirth WW, Jacobson SG, Hemo I, Sharon D. Hum Gene Ther. 2010 Dec;21(12):1749-57. doi: 10.1089/hum.2010.047. Epub 2010 Nov 3. PMID: 20604683 [PubMed – indexed for MEDLINE].
  134. Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. Boye SE, Boye SL, Pang J, Ryals R, Everhart D, Umino Y, Neeley AW, Besharse J, Barlow R, Hauswirth WW. PLoS One. 2010 Jun 25;5(6):e11306. doi: 10.1371/journal.pone.0011306.  PMID: 20593011 [PubMed – indexed for MEDLINE] Free PMC Article.  
  135. In vivo RNAi-mediated alpha-synuclein silencing induces nigrostriatal degeneration. Gorbatyuk OS, Li S, Nash K, Gorbatyuk M, Lewin AS, Sullivan LF, Mandel RJ, Chen W, Meyers C, Manfredsson FP, Muzyczka N. Mol Ther. 2010 Aug;18(8):1450-7. doi: 10.1038/mt.2010.115. Epub 2010 Jun 15. PMID: 20551914 [PubMed – indexed for MEDLINE] Free PMC Article.
  136. Mechanistic basis for the failure of cone transducin to translocate: why cones are never blinded by light. Lobanova ES, Herrmann R, Finkelstein S, Reidel B, Skiba NP, Deng WT, Jo R, Weiss ER, Hauswirth WW, Arshavsky VY. J Neurosci. 2010 May 19;30(20):6815-24. doi: 10.1523/JNEUROSCI.0613-10.2010. PMID: 20484624 [PubMed – indexed for MEDLINE] Free PMC Article.
  137. Retinal disease in Rpe65-deficient mice: comparison to human leber congenital amaurosis due to RPE65 mutations. Caruso RC, Aleman TS, Cideciyan AV, Roman AJ, Sumaroka A, Mullins CL, Boye SL, Hauswirth WW, Jacobson SG. Invest Ophthalmol Vis Sci. 2010 Oct;51(10):5304-13. doi: 10.1167/iovs.10-5559. Epub 2010 May 19. PMID: 20484585 [PubMed – indexed for MEDLINE].
  138. rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters. Beltran WA, Boye SL, Boye SE, Chiodo VA, Lewin AS, Hauswirth WW, Aguirre GD. Gene Ther. 2010 Sep;17(9):1162-74. doi: 10.1038/gt.2010.56. Epub 2010 Apr 29.  PMID: 20428215 [PubMed – indexed for MEDLINE] Free PMC Article. 
  139. Gene therapy rescues cone function in congenital achromatopsia. Komáromy AM, Alexander JJ, Rowlan JS, Garcia MM, Chiodo VA, Kaya A, Tanaka JC, Acland GM, Hauswirth WW, Aguirre GD. Hum Mol Genet. 2010 Jul 1;19(13):2581-93. doi: 10.1093/hmg/ddq136. Epub 2010 Apr 8. Erratum in: Hum Mol Genet. 2011 Dec 15;20(24):5024. PMID: 20378608 [PubMed – indexed for MEDLINE] Free PMC Article. 
  140. Achromatopsia as a potential candidate for gene therapy. Pang JJ, Alexander J, Lei B, Deng W, Zhang K, Li Q, Chang B, Hauswirth WW. Adv Exp Med Biol. 2010;664:639-46. doi: 10.1007/978-1-4419-1399-9_73. PMID: 20238068 [PubMed – indexed for MEDLINE] Free PMC Article. 
  141. Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency. Pang J, Boye SE, Lei B, Boye SL, Everhart D, Ryals R, Umino Y, Rohrer B, Alexander J, Li J, Dai X, Li Q, Chang B, Barlow R, Hauswirth WW. Gene Ther. 2010 Jul;17(7):815-26. doi: 10.1038/gt.2010.29. Epub 2010 Mar 18. PMID: 20237510 [PubMed – indexed for MEDLINE] Free PMC Article. 
  142. Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78. Gorbatyuk MS, Knox T, LaVail MM, Gorbatyuk OS, Noorwez SM, Hauswirth WW, Lin JH, Muzyczka N, Lewin AS. Proc Natl Acad Sci U S A. 2010 Mar 30;107(13):5961-6. doi: 10.1073/pnas.0911991107. Epub 2010 Mar 15. PMID: 20231467 [PubMed – indexed for MEDLINE] Free PMC Article. 
  143. Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse. Kong F, Li W, Li X, Zheng Q, Dai X, Zhou X, Boye SL, Hauswirth WW, Qu J, Pang JJ. Exp Eye Res. 2010 May;90(5):546-54. doi: 10.1016/j.exer.2010.01.011. Epub 2010 Feb 4. PMID: 20138034 [PubMed – indexed for MEDLINE] Free PMC Article. 
  144. Efficient expression of self-complementary AAV in ganglion cells of the ex vivo primate retina. Koilkonda RD, Hauswirth WW, Guy J.  Mol Vis. 2009 Dec 16;15:2796-802. PMID: 20019878 [PubMed – indexed for MEDLINE] Free PMC Article. 
  145. Selective tropism of the recombinant adeno-associated virus 9 serotype for rat cardiac tissue. Qi Y, Liu X, Li H, Shenoy V, Li Q, Hauswirth WW, Sumners C, Katovich MJ. J Gene Med. 2010 Jan;12(1):22-34. doi: 10.1002/jgm.1404. PMID: 19830780 [PubMed – indexed for MEDLINE].
  146. Functional interchangeability of rod and cone transducin alpha-subunits. Deng WT, Sakurai K, Liu J, Dinculescu A, Li J, Pang J, Min SH, Chiodo VA, Boye SL, Chang B, Kefalov VJ, Hauswirth WW. Proc Natl Acad Sci U S A. 2009 Oct 20;106(42):17681-6. doi: 10.1073/pnas.0901382106. Epub 2009 Oct 6. PMID: 19815523 [PubMed – indexed for MEDLINE] Free PMC Article. 
  147. Gene therapy for red-green colour blindness in adult primates. Mancuso K, Hauswirth WW, Li Q, Connor TB, Kuchenbecker JA, Mauck MC, Neitz J, Neitz M. Nature. 2009 Oct 8;461(7265):784-7. doi: 10.1038/nature08401. Epub 2009 Sep 16. PMID: 19759534 [PubMed – indexed for MEDLINE] Free PMC Article. 
  148. AAV retinal transduction in a large animal model species: comparison of a self-complementary AAV2/5 with a single-stranded AAV2/5 vector. Petersen-Jones SM, Bartoe JT, Fischer AJ, Scott M, Boye SL, Chiodo V, Hauswirth WW. Mol Vis. 2009 Sep 11;15:1835-42. PMID: 19756181 [PubMed – indexed for MEDLINE] Free PMC Article. 
  149. Vision 1 year after gene therapy for Leber’s congenital amaurosis. Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, Windsor EA, Conlon TJ, Sumaroka A, Roman AJ, Byrne BJ, Jacobson SG. N Engl J Med. 2009 Aug 13;361(7):725-7. doi: 10.1056/NEJMc0903652. No abstract available. PMID: 19675341 [PubMed – indexed for MEDLINE] Free PMC Article. 
  150. Delivery of antioxidant enzyme genes to protect against ischemia/reperfusion-induced injury to retinal microvasculature. Chen B, Caballero S, Seo S, Grant MB, Lewin AS. Invest Ophthalmol Vis Sci. 2009 Dec;50(12):5587-95. doi: 10.1167/iovs.09-3633. Epub 2009 Jul 23. PMID: 19628743 [PubMed – indexed for MEDLINE] Free PMC Article.
  151. Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, Windsor EA, Conlon TJ, Sumaroka A, Pang JJ, Roman AJ, Byrne BJ, Jacobson SG. Hum Gene Ther. 2009 Sep;20(9):999-1004. doi: 10.1089/hum.2009.086. PMID: 19583479 [PubMed – indexed for MEDLINE] Free PMC Article. 
  152. Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system. Guy J, Qi X, Koilkonda RD, Arguello T, Chou TH, Ruggeri M, Porciatti V, Lewin AS, Hauswirth WW. Invest Ophthalmol Vis Sci. 2009 Sep;50(9):4205-14. doi: 10.1167/iovs.08-3214. Epub 2009 Apr 22.  PMID: 19387075 [PubMed – indexed for MEDLINE] Free PMC Article.
  153. Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye. Li W, Kong F, Li X, Dai X, Liu X, Zheng Q, Wu R, Zhou X, Lü F, Chang B, Li Q, Hauswirth WW, Qu J, Pang JJ.  Mol Vis. 2009;15:267-75. Epub 2009 Feb 6. PMID: 19190735 [PubMed – indexed for MEDLINE] Free PMC Article.
  154. High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Petrs-Silva H, Dinculescu A, Li Q, Min SH, Chiodo V, Pang JJ, Zhong L, Zolotukhin S, Srivastava A, Lewin AS, Hauswirth WW. Mol Ther. 2009 Mar;17(3):463-71. doi: 10.1038/mt.2008.269. Epub 2008 Dec 16. PMID: 19066593 [PubMed – indexed for MEDLINE] Free PMC Article. 
  155. Effect of CNTF on retinal ganglion cell survival in experimental glaucoma. Pease ME, Zack DJ, Berlinicke C, Bloom K, Cone F, Wang Y, Klein RL, Hauswirth WW, Quigley HA.  Invest Ophthalmol Vis Sci. 2009 May;50(5):2194-200. doi: 10.1167/iovs.08-3013. Epub 2008 Dec 5. PMID: 19060281 [PubMed – indexed for MEDLINE] Free PMC Article. 
  156. XIAP effects on retinal detachment-induced photoreceptor apoptosis [corrected]. Zadro-Lamoureux LA, Zacks DN, Baker AN, Zheng QD, Hauswirth WW, Tsilfidis C.  Invest Ophthalmol Vis Sci. 2009 Mar;50(3):1448-53. doi: 10.1167/iovs.08-2855. Epub 2008 Dec 5. Erratum in: Invest Ophthalmol Vis Sci. 2009 Apr;50(4):1530. PMID: 19060276 [PubMed – indexed for MEDLINE] Free PMC Article.  
  157. Different tropism of adenoviruses and adeno-associated viruses to corneal cells: implications for corneal gene therapy. Liu J, Saghizadeh M, Tuli SS, Kramerov AA, Lewin AS, Bloom DC, Hauswirth WW, Castro MG, Schultz GS, Ljubimov AV. Mol Vis. 2008;14:2087-96. Epub 2008 Nov 18. PMID: 19023450 [PubMed – indexed for MEDLINE] Free PMC Article. 
  158. Angiostatin overexpression is associated with an improvement in chronic kidney injury by an anti-inflammatory mechanism. Mu W, Long DA, Ouyang X, Agarwal A, Cruz PE, Roncal CA, Nakagawa T, Yu X, Hauswirth WW, Johnson RJ. Am J Physiol Renal Physiol. 2009 Jan;296(1):F145-52. doi: 10.1152/ajprenal.90430.2008. Epub 2008 Oct 29. PMID: 18971211 [PubMed – indexed for MEDLINE] Free PMC Article. 
  159. Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential. Li Q, Miller R, Han PY, Pang J, Dinculescu A, Chiodo V, Hauswirth WW. Mol Vis. 2008 Sep 24;14:1760-9. PMID: 18836574 [PubMed – indexed for MEDLINE] Free PMC Article. 
  160. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Cideciyan AV, Aleman TS, Boye SL, Schwartz SB, Kaushal S, Roman AJ, Pang JJ, Sumaroka A, Windsor EA, Wilson JM, Flotte TR, Fishman GA, Heon E, Stone EM, Byrne BJ, Jacobson SG, Hauswirth WW. Proc Natl Acad Sci U S A. 2008 Sep 30;105(39):15112-7. doi: 10.1073/pnas.0807027105. Epub 2008 Sep 22. PMID: 18809924 [PubMed – indexed for MEDLINE] Free PMC Article.  
  161. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG. Hum Gene Ther. 2008 Oct;19(10):979-90. doi: 10.1089/hum.2008.107. PMID: 18774912 [PubMed – indexed for MEDLINE] Free PMC Article. 
  162. Downregulation of p22phox in retinal pigment epithelial cells inhibits choroidal neovascularization in mice. Li Q, Dinculescu A, Shan Z, Miller R, Pang J, Lewin AS, Raizada MK, Hauswirth WW. Mol Ther. 2008 Oct;16(10):1688-94. doi: 10.1038/mt.2008.164. Epub 2008 Jul 29. PMID: 18665154 [PubMed – indexed for MEDLINE].
  163. Novel anti-VEGF chimeric molecules delivered by AAV vectors for inhibition of retinal neovascularization. Pechan P, Rubin H, Lukason M, Ardinger J, DuFresne E, Hauswirth WW, Wadsworth SC, Scaria A. Gene Ther. 2009 Jan;16(1):10-6. doi: 10.1038/gt.2008.115. Epub 2008 Jul 17. PMID: 18633446 [PubMed – indexed for MEDLINE].
  164. Longitudinal evaluation of expression of virally delivered transgenes in gerbil cone photoreceptors. Mauck MC, Mancuso K, Kuchenbecker JA, Connor TB, Hauswirth WW, Neitz J, Neitz M. Vis Neurosci. 2008 May-Jun;25(3):273-82. doi: 10.1017/S0952523808080577. PMID: 18598398 [PubMed – indexed for MEDLINE] Free PMC Article.  
  165. Prospects for retinal cone-targeted gene therapy. Alexander JJ, Hauswirth WW. Drug News Perspect. 2008 Jun;21(5):267-71. doi: 10.1358/dnp.2008.21.5.1219012. PMID: 18596991 [PubMed – indexed for MEDLINE].
  166. AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation. Pang JJ, Boye SL, Kumar A, Dinculescu A, Deng W, Li J, Li Q, Rani A, Foster TC, Chang B, Hawes NL, Boatright JH, Hauswirth WW. Invest Ophthalmol Vis Sci. 2008 Oct;49(10):4278-83. doi: 10.1167/iovs.07-1622. Epub 2008 Jun 27. PMID: 18586879 [PubMed – indexed for MEDLINE] Free PMC Article. 
  167. Dicer inactivation leads to progressive functional and structural degeneration of the mouse retina. Damiani D, Alexander JJ, O’Rourke JR, McManus M, Jadhav AP, Cepko CL, Hauswirth WW, Harfe BD, Strettoi E. J Neurosci. 2008 May 7;28(19):4878-87. doi: 10.1523/JNEUROSCI.0828-08.2008. PMID: 18463241 [PubMed – indexed for MEDLINE] Free PMC Article. 
  168. Effect of late-stage therapy on disease progression in AAV-mediated rescue of photoreceptor cells in the retinoschisin-deficient mouse. Janssen A, Min SH, Molday LL, Tanimoto N, Seeliger MW, Hauswirth WW, Molday RS, Weber BH. Mol Ther. 2008 Jun;16(6):1010-7. doi: 10.1038/mt.2008.57. Epub 2008 Mar 25. PMID: 18388913 [PubMed – indexed for MEDLINE].
  169. Targeting gene expression to cones with human cone opsin promoters in recombinant AAV. Komáromy AM, Alexander JJ, Cooper AE, Chiodo VA, Glushakova LG, Acland GM, Hauswirth WW, Aguirre GD. Gene Ther. 2008 Jul;15(14):1049-55. doi: 10.1038/gt.2008.32. Epub 2008 Mar 13. Erratum in: Gene Ther. 2011 Dec;18(12):1179. Gene Ther. 2008 Jul;15(14):1073. Glushakova, L G [added]. PMID: 18337838 [PubMed – indexed for MEDLINE] Free PMC Article.
  170. Toward a higher fidelity model of AMD. Raisler BJ, Nozaki M, Baffi J, Hauswirth WW, Ambati J. Adv Exp Med Biol. 2008;613:185-92. doi: 10.1007/978-0-387-74904-4_21. No abstract available. PMID: 18188944 [PubMed – indexed for MEDLINE].
  171. Adeno-associated viral vectors and the retina. Alexander JJ, Hauswirth WW. Adv Exp Med Biol. 2008;613:121-8. doi: 10.1007/978-0-387-74904-4_13. Review. PMID: 18188936 [PubMed – indexed for MEDLINE].
  172. Gene therapy for mouse models of ADRP. Gorbatyuk MS, Hauswirth WW, Lewin AS. Adv Exp Med Biol. 2008;613:107-12. doi: 10.1007/978-0-387-74904-4_11. No abstract available. PMID: 18188934 [PubMed – indexed for MEDLINE].
  173. Unexpected off-targeting effects of anti-huntingtin ribozymes and siRNA in vivo. Denovan-Wright EM, Rodriguez-Lebron E, Lewin AS, Mandel RJ. Neurobiol Dis. 2008 Mar;29(3):446-55. doi: 10.1016/j.nbd.2007.11.003. Epub 2007 Nov 12. PMID: 18166484 [PubMed – indexed for MEDLINE] Free PMC Article. 
  174. Rapid, widespread transduction of the murine myocardium using self-complementary Adeno-associated virus. Andino LM, Conlon TJ, Porvasnik SL, Boye SL, Hauswirth WW, Lewin AS. Genet Vaccines Ther. 2007 Dec 10;5:13. PMID: 18070352 [PubMed] Free PMC Article. 
  175. AAV-mediated knockdown of phospholamban leads to improved contractility and calcium handling in cardiomyocytes. Andino LM, Takeda M, Kasahara H, Jakymiw A, Byrne BJ, Lewin AS. J Gene Med. 2008 Feb;10(2):132-42. PMID: 18064719 [PubMed – indexed for MEDLINE].
  176. Intraocular CNTF reduces vision in normal rats in a dose-dependent manner. McGill TJ, Prusky GT, Douglas RM, Yasumura D, Matthes MT, Nune G, Donohue-Rolfe K, Yang H, Niculescu D, Hauswirth WW, Girman SV, Lund RD, Duncan JL, LaVail MM. Invest Ophthalmol Vis Sci. 2007 Dec;48(12):5756-66. PMID: 18055829 [PubMed – indexed for MEDLINE].
  177. Long-term suppression of neurodegeneration in chronic experimental optic neuritis: antioxidant gene therapy. Qi X, Sun L, Lewin AS, Hauswirth WW, Guy J. Invest Ophthalmol Vis Sci. 2007 Dec;48(12):5360-70. PMID: 18055782 [PubMed – indexed for MEDLINE].
  178. Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis. Roman AJ, Boye SL, Aleman TS, Pang JJ, McDowell JH, Boye SE, Cideciyan AV, Jacobson SG, Hauswirth WW. Mol Vis. 2007 Sep 18;13:1701-10. PMID: 17960108 [PubMed – indexed for MEDLINE].
  179. Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: effects of serotype and site of administration. Pang JJ, Lauramore A, Deng WT, Li Q, Doyle TJ, Chiodo V, Li J, Hauswirth WW. Vision Res. 2008 Feb;48(3):377-85. Epub 2007 Oct 22. PMID: 17950399 [PubMed – indexed for MEDLINE] Free Article. 
  180. Cone-specific expression using a human red opsin promoter in recombinant AAV. Li Q, Timmers AM, Guy J, Pang J, Hauswirth WW. Vision Res. 2008 Feb;48(3):332-8. Epub 2007 Oct 1. PMID: 17905404 [PubMed – indexed for MEDLINE] Free Article. 
  181. SOD2 knockdown mouse model of early AMD. Justilien V, Pang JJ, Renganathan K, Zhan X, Crabb JW, Kim SR, Sparrow JR, Hauswirth WW, Lewin AS. Invest Ophthalmol Vis Sci. 2007 Oct;48(10):4407-20. PMID: 17898259 [PubMed – indexed for MEDLINE].
  182. Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation. Aguirre GK, Komáromy AM, Cideciyan AV, Brainard DH, Aleman TS, Roman AJ, Avants BB, Gee JC, Korczykowski M, Hauswirth WW, Acland GM, Aguirre GD, Jacobson SG. PLoS Med. 2007 Jun;4(6):e230. PMID: 17594175 [PubMed – indexed for MEDLINE] Free PMC Article. 
  183. Restoration of cone vision in a mouse model of achromatopsia. Alexander JJ, Umino Y, Everhart D, Chang B, Min SH, Li Q, Timmers AM, Hawes NL, Pang JJ, Barlow RB, Hauswirth WW. Nat Med. 2007 Jun;13(6):685-7. Epub 2007 May 21. PMID: 17515894 [PubMed – indexed for MEDLINE] Free PMC Article. 
  184. Brain-derived neurotrophic factor gene transfer with adeno-associated viral and lentiviral vectors prevents rubrospinal neuronal atrophy and stimulates regeneration-associated gene expression after acute cervical spinal cord injury. Kwon BK, Liu J, Lam C, Plunet W, Oschipok LW, Hauswirth W, Di Polo A, Blesch A, Tetzlaff W. Spine (Phila Pa 1976). 2007 May 15;32(11):1164-73. PMID: 17495772 [PubMed – indexed for MEDLINE].
  185. Anti-clarin-1 AAV-delivered ribozyme induced apoptosis in the mouse cochlea. Aarnisalo AA, Pietola L, Joensuu J, Isosomppi J, Aarnisalo P, Dinculescu A, Lewin AS, Flannery J, Hauswirth WW, Sankila EM, Jero J. Hear Res. 2007 Aug;230(1-2):9-16. Epub 2007 Apr 5. PMID: 17493778 [PubMed – indexed for MEDLINE].
  186. Increased sensitivity to light-induced damage in a mouse model of autosomal dominant retinal disease. White DA, Fritz JJ, Hauswirth WW, Kaushal S, Lewin AS. Invest Ophthalmol Vis Sci. 2007 May;48(5):1942-51. Erratum in: Invest Ophthalmol Vis Sci. 2007 Aug;48(8):3436. Fritz, Jason J. (added).  PMID: 17460245 [PubMed – indexed for MEDLINE].
  187. Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina. Mancuso K, Hendrickson AE, Connor TB Jr, Mauck MC, Kinsella JJ, Hauswirth WW, Neitz J, Neitz M. J Opt Soc Am A Opt Image Sci Vis. 2007 May;24(5):1411-6. PMID: 17429487 [PubMed – indexed for MEDLINE].
  188. Down-regulation of expression of rat pyruvate dehydrogenase E1alpha gene by self-complementary adeno-associated virus-mediated small interfering RNA delivery. Han Z, Gorbatyuk M, Thomas J Jr, Lewin AS, Srivastava A, Stacpoole PW. Mitochondrion. 2007 Jul;7(4):253-9. Epub 2007 Feb 20. PMID: 17392036 [PubMed – indexed for MEDLINE] Free PMC Article.  
  189. XIAP protection of photoreceptors in animal models of retinitis pigmentosa. Leonard KC, Petrin D, Coupland SG, Baker AN, Leonard BC, LaCasse EC, Hauswirth WW, Korneluk RG, Tsilfidis C. PLoS One. 2007 Mar 21;2(3):e314. PMID: 17375200 [PubMed – indexed for MEDLINE] Free PMC Article. 
  190. Molecular and cellular alterations induced by sustained expression of ciliary neurotrophic factor in a mouse model of retinitis pigmentosa. Rhee KD, Ruiz A, Duncan JL, Hauswirth WW, Lavail MM, Bok D, Yang XJ. Invest Ophthalmol Vis Sci. 2007 Mar;48(3):1389-400.  PMID: 17325188 [PubMed – indexed for MEDLINE].
  191. Use of mitochondrial antioxidant defenses for rescue of cells with a Leber hereditary optic neuropathy-causing mutation. Qi X, Sun L, Hauswirth WW, Lewin AS, Guy J. Arch Ophthalmol. 2007 Feb;125(2):268-72. PMID: 17296905 [PubMed – indexed for MEDLINE].
  192. Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery. Gorbatyuk M, Justilien V, Liu J, Hauswirth WW, Lewin AS. Vision Res. 2007 Apr;47(9):1202-8. Epub 2007 Feb 12. PMID: 17292939 [PubMed – indexed for MEDLINE] Free PMC Article. 
  193. Suppression of mitochondrial oxidative stress provides long-term neuroprotection in experimental optic neuritis. Qi X, Lewin AS, Sun L, Hauswirth WW, Guy J. Invest Ophthalmol Vis Sci. 2007 Feb;48(2):681-91. PMID: 17251466 [PubMed – indexed for MEDLINE].
  194. Disease mechanisms and gene therapy in a mouse model for X-linked retinoschisis. Molday LL, Min SH, Seeliger MW, Wu WW, Dinculescu A, Timmers AM, Janssen A, Tonagel F, Hudl K, Weber BH, Hauswirth WW, Molday RS. Adv Exp Med Biol. 2006;572:283-9. No abstract available. PMID: 17249585 [PubMed – indexed for MEDLINE].
  195. Down-regulation of rhodopsin gene expression by AAV-vectored short interfering RNA. Teusner JT, Lewin AS, Hauswirth WW. Adv Exp Med Biol. 2006;572:233-8. No abstract available. PMID: 17249579 [PubMed – indexed for MEDLINE].
  196. Dual gene therapy with extracellular superoxide dismutase and catalase attenuates experimental optic neuritis. Qi X, Hauswirth WW, Guy J. Mol Vis. 2007 Jan 5;13:1-11.  PMID: 17242675 [PubMed – indexed for MEDLINE] Free PMC Article.
  197. The mutant human ND4 subunit of complex I induces optic neuropathy in the mouse. Qi X, Sun L, Lewin AS, Hauswirth WW, Guy J. Invest Ophthalmol Vis Sci. 2007 Jan;48(1):1-10. PMID: 17197509 [PubMed – indexed for MEDLINE].
  198. Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme. Gorbatyuk M, Justilien V, Liu J, Hauswirth WW, Lewin AS. Exp Eye Res. 2007 Jan;84(1):44-52. Epub 2006 Nov 1. PMID: 17083931 [PubMed – indexed for MEDLINE] Free PMC Article. 
  199. Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Jacobson SG, Boye SL, Aleman TS, Conlon TJ, Zeiss CJ, Roman AJ, Cideciyan AV, Schwartz SB, Komaromy AM, Doobrajh M, Cheung AY, Sumaroka A, Pearce-Kelling SE, Aguirre GD, Kaushal S, Maguire AM, Flotte TR, Hauswirth WW. Hum Gene Ther. 2006 Aug;17(8):845-58. PMID: 16942444 [PubMed – indexed for MEDLINE].
  200. Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1. Haire SE, Pang J, Boye SL, Sokal I, Craft CM, Palczewski K, Hauswirth WW, Semple-Rowland SL. Invest Ophthalmol Vis Sci. 2006 Sep;47(9):3745-53. PMID: 16936082 [PubMed – indexed for MEDLINE] Free PMC Article. 
  201. Mitochondrial protein nitration primes neurodegeneration in experimental autoimmune encephalomyelitis. Qi X, Lewin AS, Sun L, Hauswirth WW, Guy J. J Biol Chem. 2006 Oct 20;281(42):31950-62. Epub 2006 Aug 18. PMID: 16920708 [PubMed – indexed for MEDLINE] Free Article. 
  202. Human blue-opsin promoter preferentially targets reporter gene expression to rat s-cone photoreceptors. Glushakova LG, Timmers AM, Pang J, Teusner JT, Hauswirth WW. Invest Ophthalmol Vis Sci. 2006 Aug;47(8):3505-13. PMID: 16877422 [PubMed – indexed for MEDLINE].
  203. Cortical visual function in the rd12 mouse model of Leber Congenital Amarousis (LCA) after gene replacement therapy to restore retinal function. Nusinowitz S, Ridder WH 3rd, Pang JJ, Chang B, Noorwez SM, Kaushal S, Hauswirth WW, Heckenlively JR. Vision Res. 2006 Oct;46(22):3926-34. Epub 2006 Jul 11. PMID: 16814838 [PubMed – indexed for MEDLINE] Free Article. 
  204. Proceedings of the Third International Symposium on Retinopathy of Prematurity: an update on ROP from the lab to the nursery (November 2003, Anaheim, California). Lutty GA, Chan-Ling T, Phelps DL, Adamis AP, Berns KI, Chan CK, Cole CH, D’Amore PA, Das A, Deng WT, Dobson V, Flynn JT, Friedlander M, Fulton A, Good WV, Grant MB, Hansen R, Hauswirth WW, Hardy RJ, Hinton DR, Hughes S, McLeod DS, Palmer EA, Patz A, Penn JS, Raisler BJ, Repka MX, Saint-Geniez M, Shaw LC, Shima DT, Smith BT, Smith LE, Tahija SG, Tasman W, Trese MT. Mol Vis. 2006 May 23;12:532-80. PMID: 16735995 [PubMed – indexed for MEDLINE] Free Article. 
  205. Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Jacobson SG, Acland GM, Aguirre GD, Aleman TS, Schwartz SB, Cideciyan AV, Zeiss CJ, Komaromy AM, Kaushal S, Roman AJ, Windsor EA, Sumaroka A, Pearce-Kelling SE, Conlon TJ, Chiodo VA, Boye SL, Flotte TR, Maguire AM, Bennett J, Hauswirth WW. Mol Ther. 2006 Jun;13(6):1074-84. Epub 2006 Apr 27. PMID: 16644289 [PubMed – indexed for MEDLINE].
  206. Does recombinant adeno-associated virus-vectored proximal region of mouse rhodopsin promoter support only rod-type specific expression in vivo? Glushakova LG, Timmers AM, Issa TM, Cortez NG, Pang J, Teusner JT, Hauswirth WW. Mol Vis. 2006 Apr 7;12:298-309. PMID: 16617297 [PubMed – indexed for MEDLINE] Free Article. 
  207. Extracellular signal-regulated kinases 1/2 are required for adult retinal ganglion cell axon regeneration induced by fibroblast growth factor-2. Sapieha PS, Hauswirth WW, Di Polo A. J Neurosci Res. 2006 May 1;83(6):985-95. PMID: 16493686 [PubMed – indexed for MEDLINE].
  208. The consortium project to treat RPE65 deficiency in humans. Hauswirth WW. Retina. 2005 Dec;25(8 Suppl):S60. Review. No abstract available. PMID: 16374340 [PubMed – indexed for MEDLINE].
  209. XIAP-mediated neuroprotection in retinal ischemia. Renwick J, Narang MA, Coupland SG, Xuan JY, Baker AN, Brousseau J, Petrin D, Munger R, Leonard BC, Hauswirth WW, Korneluk RG, Tsilfidis C. Gene Ther. 2006 Feb;13(4):339-47. PMID: 16307001 [PubMed – indexed for MEDLINE].
  210. Adeno-associated virus-mediated expression of vascular endothelial growth factor peptides inhibits retinal neovascularization in a mouse model of oxygen-induced retinopathy. Deng WT, Yan Z, Dinculescu A, Pang J, Teusner JT, Cortez NG, Berns KI, Hauswirth WW. Hum Gene Ther. 2005 Nov;16(11):1247-54. PMID: 16259558 [PubMed – indexed for MEDLINE].
  211. IL-10 suppresses chemokines, inflammation, and fibrosis in a model of chronic renal disease. Mu W, Ouyang X, Agarwal A, Zhang L, Long DA, Cruz PE, Roncal CA, Glushakova OY, Chiodo VA, Atkinson MA, Hauswirth WW, Flotte TR, Rodriguez-Iturbe B, Johnson RJ. J Am Soc Nephrol. 2005 Dec;16(12):3651-60. Epub 2005 Oct 26. PMID: 16251240 [PubMed – indexed for MEDLINE] Free Article. 
  212. Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis. Batten ML, Imanishi Y, Tu DC, Doan T, Zhu L, Pang J, Glushakova L, Moise AR, Baehr W, Van Gelder RN, Hauswirth WW, Rieke F, Palczewski K. PLoS Med. 2005 Nov;2(11):e333. Epub 2005 Nov 1. PMID: 16250670 [PubMed – indexed for MEDLINE] Free PMC Article. 
  213. Gene therapy for autosomal dominant disorders of keratin. Lewin AS, Glazer PM, Milstone LM. J Investig Dermatol Symp Proc. 2005 Oct;10(1):47-61. Review. PMID: 16250209 [PubMed – indexed for MEDLINE] Free Article. 
  214. Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness. Acland GM, Aguirre GD, Bennett J, Aleman TS, Cideciyan AV, Bennicelli J, Dejneka NS, Pearce-Kelling SE, Maguire AM, Palczewski K, Hauswirth WW, Jacobson SG. Mol Ther. 2005 Dec;12(6):1072-82. Epub 2005 Oct 14. PMID: 16226919 [PubMed – indexed for MEDLINE] Free PMC Article. 
  215. Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis. Pang JJ, Chang B, Kumar A, Nusinowitz S, Noorwez SM, Li J, Rani A, Foster TC, Chiodo VA, Doyle T, Li H, Malhotra R, Teusner JT, McDowell JH, Min SH, Li Q, Kaushal S, Hauswirth WW. Mol Ther. 2006 Mar;13(3):565-72. Epub 2005 Oct 11. PMID: 16223604 [PubMed – indexed for MEDLINE].
  216. Ribozyme knockdown of the gamma-subunit of rod cGMP phosphodiesterase alters the ERG and retinal morphology in wild-type mice. Liu J, Timmers AM, Lewin AS, Hauswirth WW. Invest Ophthalmol Vis Sci. 2005 Oct;46(10):3836-44. PMID: 16186371 [PubMed – indexed for MEDLINE].
  217. Knockdown of wild-type mouse rhodopsin using an AAV vectored ribozyme as part of an RNA replacement approach. Gorbatyuk MS, Pang JJ, Thomas J Jr, Hauswirth WW, Lewin AS. Mol Vis. 2005 Aug 29;11:648-56. PMID: 16145542 [PubMed – indexed for MEDLINE] Free Article. 
  218. Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis. Min SH, Molday LL, Seeliger MW, Dinculescu A, Timmers AM, Janssen A, Tonagel F, Tanimoto N, Weber BH, Molday RS, Hauswirth WW. Mol Ther. 2005 Oct;12(4):644-51. PMID: 16027044 [PubMed – indexed for MEDLINE].
  219. Localized gene expression following administration of adeno-associated viral vectors via pancreatic ducts. Loiler SA, Tang Q, Clarke T, Campbell-Thompson ML, Chiodo V, Hauswirth W, Cruz P, Perret-Gentil M, Atkinson MA, Ramiya VK, Flotte TR. Mol Ther. 2005 Sep;12(3):519-27. PMID: 15979413 [PubMed – indexed for MEDLINE].
  220. Activation of the extracellular signal-regulated kinase 1/2 pathway by AAV gene transfer protects retinal ganglion cells in glaucoma. Zhou Y, Pernet V, Hauswirth WW, Di Polo A. Mol Ther. 2005 Sep;12(3):402-12. PMID: 15975850 [PubMed – indexed for MEDLINE].
  221. Adeno-associated virus-vectored gene therapy for retinal disease. Dinculescu A, Glushakova L, Min SH, Hauswirth WW. Hum Gene Ther. 2005 Jun;16(6):649-63. Review. PMID: 15960597 [PubMed – indexed for MEDLINE].
  222. Interleukin 10 attenuates neointimal proliferation and inflammation in aortic allografts by a heme oxygenase-dependent pathway. Chen S, Kapturczak MH, Wasserfall C, Glushakova OY, Campbell-Thompson M, Deshane JS, Joseph R, Cruz PE, Hauswirth WW, Madsen KM, Croker BP, Berns KI, Atkinson MA, Flotte TR, Tisher CC, Agarwal A. Proc Natl Acad Sci U S A. 2005 May 17;102(20):7251-6. Epub 2005 May 6. PMID: 15878989 [PubMed – indexed for MEDLINE] Free PMC Article. 
  223. Extracellular signal-regulated kinase 1/2 mediates survival, but not axon regeneration, of adult injured central nervous system neurons in vivo. Pernet V, Hauswirth WW, Di Polo A. J Neurochem. 2005 Apr;93(1):72-83. PMID: 15773907 [PubMed – indexed for MEDLINE] Free Article.
  224. Modulation of the expression of the transcription factor Max in rat retinal ganglion cells by a recombinant adeno-associated viral vector. Petrs-Silva H, Chiodo V, Chiarini LB, Hauswirth WW, Linden R. Braz J Med Biol Res. 2005 Mar;38(3):375-9. Epub 2005 Mar 8. Erratum in: Braz J Med Biol Res. 2005 Jun;38(6):977. PMID: 15761617 [PubMed – indexed for MEDLINE] Free Article. 
  225. Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. Chen S, Kapturczak M, Loiler SA, Zolotukhin S, Glushakova OY, Madsen KM, Samulski RJ, Hauswirth WW, Campbell-Thompson M, Berns KI, Flotte TR, Atkinson MA, Tisher CC, Agarwal A. Hum Gene Ther. 2005 Feb;16(2):235-47. PMID: 15761263 [PubMed – indexed for MEDLINE] Free PMC Article.
  226. SDF-1 is both necessary and sufficient to promote proliferative retinopathy. Butler JM, Guthrie SM, Koc M, Afzal A, Caballero S, Brooks HL, Mames RN, Segal MS, Grant MB, Scott EW. J Clin Invest. 2005 Jan;115(1):86-93. PMID: 15630447 [PubMed – indexed for MEDLINE] Free PMC Article. 
  227. SOD2 gene transfer protects against optic neuropathy induced by deficiency of complex I. Qi X, Lewin AS, Sun L, Hauswirth WW, Guy J. Ann Neurol. 2004 Aug;56(2):182-91. PMID: 15293270 [PubMed – indexed for MEDLINE].
  228. AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice. Hennig AK, Ogilvie JM, Ohlemiller KK, Timmers AM, Hauswirth WW, Sands MS. Mol Ther. 2004 Jul;10(1):106-16. PMID: 15233947 [PubMed – indexed for MEDLINE].
  229. XIAP protects photoreceptors from n-methyl-n-nitrosourea-induced retinal degeneration. Petrin D, Baker A, Brousseau J, Coupland S, Liston P, Hauswirth WW, Komeluk RG, Tsilfidis C. Adv Exp Med Biol. 2003;533:385-93. No abstract available. PMID: 15180289 [PubMed – indexed for MEDLINE].
  230. Design and validation of therapeutic hammerhead ribozymes for autosomal dominant diseases. Fritz JJ, Gorbatyuk M, Lewin AS, Hauswirth WW. Methods Mol Biol. 2004;252:221-36. PMID: 15017052 [PubMed – indexed for MEDLINE].
  231. Range of retinal diseases potentially treatable by AAV-vectored gene therapy. Hauswirth WW, Li Q, Raisler B, Timmers AM, Berns KI, Flannery JG, LaVail MM, Lewin AS. Novartis Found Symp. 2004;255:179-88; discussion 188-94. Review. PMID: 14750604 [PubMed – indexed for MEDLINE].
  232. Gene therapy with brain-derived neurotrophic factor as a protection: retinal ganglion cells in a rat glaucoma model. Martin KR, Quigley HA, Zack DJ, Levkovitch-Verbin H, Kielczewski J, Valenta D, Baumrind L, Pease ME, Klein RL, Hauswirth WW. Invest Ophthalmol Vis Sci. 2003 Oct;44(10):4357-65. PMID: 14507880 [PubMed – indexed for MEDLINE].
  233. Decreased expression of the insulin-like growth factor 1 receptor by ribozyme cleavage. Shaw LC, Afzal A, Lewin AS, Timmers AM, Spoerri PE, Grant MB. Invest Ophthalmol Vis Sci. 2003 Sep;44(9):4105-13. PMID: 12939334 [PubMed – indexed for MEDLINE].
  234. Anti-apoptotic effects of CNTF gene transfer on photoreceptor degeneration in experimental antibody-induced retinopathy. Adamus G, Sugden B, Shiraga S, Timmers AM, Hauswirth WW. J Autoimmun. 2003 Sep;21(2):121-9. Erratum in: J Autoimmun. 2003 Dec;21(4):393. PMID: 12935781 [PubMed – indexed for MEDLINE].
  235. Structural and functional protection of photoreceptors from MNU-induced retinal degeneration by the X-linked inhibitor of apoptosis. Petrin D, Baker A, Coupland SG, Liston P, Narang M, Damji K, Leonard B, Chiodo VA, Timmers A, Hauswirth W, Korneluk RG, Tsilfidis C. Invest Ophthalmol Vis Sci. 2003 Jun;44(6):2757-63. PMID: 12766084 [PubMed – indexed for MEDLINE].
  236. Gene delivery in renal tubular epithelial cells using recombinant adeno-associated viral vectors. Chen S, Agarwal A, Glushakova OY, Jorgensen MS, Salgar SK, Poirier A, Flotte TR, Croker BP, Madsen KM, Atkinson MA, Hauswirth WW, Berns KI, Tisher CC. J Am Soc Nephrol. 2003 Apr;14(4):947-58. PMID: 12660329 [PubMed – indexed for MEDLINE] Free Article. 
  237. Optic neuropathy induced by reductions in mitochondrial superoxide dismutase. Qi X, Lewin AS, Hauswirth WW, Guy J. Invest Ophthalmol Vis Sci. 2003 Mar;44(3):1088-96. PMID: 12601034 [PubMed – indexed for MEDLINE].
  238. Suppression of complex I gene expression induces optic neuropathy. Qi X, Lewin AS, Hauswirth WW, Guy J. Ann Neurol. 2003 Feb;53(2):198-205. PMID: 12557286 [PubMed – indexed for MEDLINE].
  239. Development of hammerhead ribozymes to modulate endogenous gene expression for functional studies. Fritz JJ, Lewin A, Hauswirth W, Agarwal A, Grant M, Shaw L. Methods. 2002 Oct;28(2):276-85. PMID: 12413427 [PubMed – indexed for MEDLINE].
  240. Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Zolotukhin S, Potter M, Zolotukhin I, Sakai Y, Loiler S, Fraites TJ Jr, Chiodo VA, Phillipsberg T, Muzyczka N, Hauswirth WW, Flotte TR, Byrne BJ, Snyder RO. Methods. 2002 Oct;28(2):158-67. PMID: 12413414 [PubMed – indexed for MEDLINE].
  241. Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy. Guy J, Qi X, Pallotti F, Schon EA, Manfredi G, Carelli V, Martinuzzi A, Hauswirth WW, Lewin AS. Ann Neurol. 2002 Nov;52(5):534-42. PMID: 12402249 [PubMed – indexed for MEDLINE].
  242. Effects of adeno-associated virus-vectored ciliary neurotrophic factor on retinal structure and function in mice with a P216L rds/peripherin mutation. Bok D, Yasumura D, Matthes MT, Ruiz A, Duncan JL, Chappelow AV, Zolutukhin S, Hauswirth W, LaVail MM. Exp Eye Res. 2002 Jun;74(6):719-35. PMID: 12126945 [PubMed – indexed for MEDLINE].
  243. Adeno-associated virus type-2 expression of pigmented epithelium-derived factor or Kringles 1-3 of angiostatin reduce retinal neovascularization. Raisler BJ, Berns KI, Grant MB, Beliaev D, Hauswirth WW. Proc Natl Acad Sci U S A. 2002 Jun 25;99(13):8909-14. Epub 2002 Jun 18. PMID: 12072560 [PubMed – indexed for MEDLINE] Free PMC Article. 
  244. AAV-mediated gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization. Mori K, Gehlbach P, Yamamoto S, Duh E, Zack DJ, Li Q, Berns KI, Raisler BJ, Hauswirth WW, Campochiaro PA. Invest Ophthalmol Vis Sci. 2002 Jun;43(6):1994-2000. PMID: 12037010 [PubMed – indexed for MEDLINE].
  245. Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model. McKinnon SJ, Lehman DM, Tahzib NG, Ransom NL, Reitsamer HA, Liston P, LaCasse E, Li Q, Korneluk RG, Hauswirth WW. Mol Ther. 2002 Jun;5(6):780-7. PMID: 12027563 [PubMed – indexed for MEDLINE] Free Article. 
  246. TrkB gene transfer protects retinal ganglion cells from axotomy-induced death in vivo. Cheng L, Sapieha P, Kittlerova P, Hauswirth WW, Di Polo A. J Neurosci. 2002 May 15;22(10):3977-86. PMID: 12019317 [PubMed – indexed for MEDLINE] Free Article. 
  247. Designing and characterizing hammerhead ribozymes for use in AAV vector-mediated retinal gene therapies. Fritz JJ, White DA, Lewin AS, Hauswirth WW. Methods Enzymol. 2002;346:358-77. No abstract available. PMID: 11883079 [PubMed – indexed for MEDLINE].
  248. Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa. McGee Sanftner LH, Abel H, Hauswirth WW, Flannery JG. Mol Ther. 2001 Dec;4(6):622-9. PMID: 11735347 [PubMed – indexed for MEDLINE] Free Article.
  249. Gene therapy restores vision in a canine model of childhood blindness. Acland GM, Aguirre GD, Ray J, Zhang Q, Aleman TS, Cideciyan AV, Pearce-Kelling SE, Anand V, Zeng Y, Maguire AM, Jacobson SG, Hauswirth WW, Bennett J. Nat Genet. 2001 May;28(1):92-5. PMID: 11326284 [PubMed – indexed for MEDLINE].
  250. Ribozyme gene therapy: applications for molecular medicine. Lewin AS, Hauswirth WW. Trends Mol Med. 2001 May;7(5):221-8. Review. PMID: 11325634 [PubMed – indexed for MEDLINE].
  251. An allele-specific hammerhead ribozyme gene therapy for a porcine model of autosomal dominant retinitis pigmentosa. Shaw LC, Skold A, Wong F, Petters R, Hauswirth WW, Lewin AS. Mol Vis. 2001 Jan 26;7:6-13. PMID: 11172137 [PubMed – indexed for MEDLINE] Free Article.
  252. Inhibition of gene expression by ribozymes. Hauswirth WW, Shaw LC, Whalen PO, Fritz JJ, White DA, Lewin AS. Methods Mol Med. 2001;47:105-24. doi: 10.1385/1-59259-085-3:105. PMID: 21394581 [PubMed].
  253. Recombinant adeno-associated virus vector-based gene transfer for defects in oxidative metabolism. Owen R IV, Lewin AP, Peel A, Wang J, Guy J, Hauswirth WW, Stacpoole PW, Flotte TR. Hum Gene Ther. 2000 Oct 10;11(15):2067-78. PMID: 11044909 [PubMed – indexed for MEDLINE].
  254. Ribozyme uses in retinal gene therapy. Hauswirth WW, Lewin AS. Prog Retin Eye Res. 2000 Nov;19(6):689-710. Review. PMID: 11029552 [PubMed – indexed for MEDLINE].
  255. Ribozyme rescue of photoreceptor cells in P23H transgenic rats: long-term survival and late-stage therapy. LaVail MM, Yasumura D, Matthes MT, Drenser KA, Flannery JG, Lewin AS, Hauswirth WW. Proc Natl Acad Sci U S A. 2000 Oct 10;97(21):11488-93. PMID: 11005848 [PubMed – indexed for MEDLINE] Free PMC Article. 
  256. Ocular gene therapy: quo vadis? Hauswirth WW, Beaufrere L. Invest Ophthalmol Vis Sci. 2000 Sep;41(10):2821-6. Review. No abstract available. PMID: 10967033 [PubMed – indexed for MEDLINE].
  257. Ribozyme gene therapy for autosomal dominant retinal disease. Hauswirth WW, LaVail MM, Flannery JG, Lewin AS. Clin Chem Lab Med. 2000 Feb;38(2):147-53. PMID: 10834402 [PubMed – indexed for MEDLINE].
  258. Ribozymes in treatment of inherited retinal disease. Shaw LC, Whalen PO, Drenser KA, Yan W, Hauswirth WW, Lewin AS. Methods Enzymol. 2000;316:761-76. No abstract available. PMID: 10800713 [PubMed – indexed for MEDLINE].
  259. Production and purification of recombinant adeno-associated virus. Hauswirth WW, Lewin AS, Zolotukhin S, Muzyczka N. Methods Enzymol. 2000;316:743-61. No abstract available. PMID: 10800712 [PubMed – indexed for MEDLINE].
  260. The eyes have it. Hauswirth WW, Timmers AM. Mol Med Today. 2000 Feb;6(2):51. No abstract available. PMID: 10681166 [PubMed – indexed for MEDLINE].
  261. Adenoviral gene therapy with catalase suppresses experimental optic neuritis. Guy J, Qi X, Wang H, Hauswirth WW. Arch Ophthalmol. 1999 Nov;117(11):1533-9. PMID: 10565523 [PubMed – indexed for MEDLINE].
  262. Reporter expression persists 1 year after adeno-associated virus-mediated gene transfer to the optic nerve. Guy J, Qi X, Muzyczka N, Hauswirth WW. Arch Ophthalmol. 1999 Jul;117(7):929-37. PMID: 10408459 [PubMed – indexed for MEDLINE].
  263. Adeno-associated viral-mediated catalase expression suppresses optic neuritis in experimental allergic encephalomyelitis. Guy J, Qi X, Hauswirth WW. Proc Natl Acad Sci U S A. 1998 Nov 10;95(23):13847-52. PMID: 9811889 [PubMed – indexed for MEDLINE] Free PMC Article. 
  264. Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa. Lewin AS, Drenser KA, Hauswirth WW, Nishikawa S, Yasumura D, Flannery JG, LaVail MM. Nat Med. 1998 Aug;4(8):967-71. Erratum in: Nat Med 1998 Sep;4(9):1081. PMID: 9701253 [PubMed – indexed for MEDLINE].
  265. Retinal gene therapy 1998: summary of a workshop. Hauswirth WW, McInnes RR. Mol Vis. 1998 Jul 21;4:11. PMID: 9675216 [PubMed – indexed for MEDLINE] Free Article. 
  266. Ribozyme-targeted destruction of RNA associated with autosomal-dominant retinitis pigmentosa. Drenser KA, Timmers AM, Hauswirth WW, Lewin AS. Invest Ophthalmol Vis Sci. 1998 Apr;39(5):681-9. PMID: 9538873 [PubMed – indexed for MEDLINE].
  267. Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Flannery JG, Zolotukhin S, Vaquero MI, LaVail MM, Muzyczka N, Hauswirth WW. Proc Natl Acad Sci U S A. 1997 Jun 24;94(13):6916-21. PMID: 9192666 [PubMed – indexed for MEDLINE] Free PMC Article. 
  268. A “humanized” green fluorescent protein cDNA adapted for high-level expression in mammalian cells. Zolotukhin S, Potter M, Hauswirth WW, Guy J, Muzyczka N. J Virol. 1996 Jul;70(7):4646-54. PMID: 8676491 [PubMed – indexed for MEDLINE] Free PMC Article. 
  269. Submitochondrial localization, cell-free synthesis, and mitochondrial import of 2-isopropylmalate synthase of yeast. Hampsey DM, Lewin AS, Kohlhaw GB. Proc Natl Acad Sci U S A. 1983 Mar;80(5):1270-4. PMID: 6338500 [PubMed – indexed for MEDLINE] Free PMC Article. 
  270. 270.Mitochondrial DNA copy number in bovine oocytes and somatic cells. Michaels GS, Hauswirth WW, Laipis PJ. Dev Biol. 1982 Nov;94(1):246-51. No abstract available. PMID: 6295849 [PubMed – indexed for MEDLINE].
  271. Adeno-associated virus DNA replication: nonunit-length molecules. Hauswirth WW, Berns KI. Virology. 1979 Feb;93(1):57-68. No abstract available. PMID: 219605 [PubMed – indexed for MEDLINE].
  272. Adeno-associated viruses. Berns KI, Hauswirth WW. Adv Virus Res. 1979;25:407-49. Review. No abstract available. PMID: 230727 [PubMed – indexed for MEDLINE].
  273. Adeno-associated virus DNA replication. Berns KI, Hauswirth WW, Fife KH, Lusby E. Cold Spring Harb Symp Quant Biol. 1979;43 Pt 2:781-7. No abstract available. PMID: 226321 [PubMed – indexed for MEDLINE].
  274. Evidence for two nucleotide sequence orientations within the terminal repetition of adeno-associated virus DNA. Spear IS, Fife KH, Hauswirth WW, Jones CJ, Berns KI. J Virol. 1977 Nov;24(2):627-34. PMID: 916029 [PubMed – indexed for MEDLINE] Free PMC Article. 
  275. Origin and termination of adeno-associated virus DNA replication. Hauswirth WW, Berns KI. Virology. 1977 May 15;78(2):488-99. No abstract available. PMID: 867815 [PubMed – indexed for MEDLINE].