William W. Hauswirth, PhD - Publications

Affiliations: 
Ophthalmology University of Florida, Gainesville, Gainesville, FL, United States 

222 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any innacuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2018 Hanke-Gogokhia C, Chiodo VA, Hauswirth WW, Frederick JM, Baehr W. Rescue of cone function in cone-only knockout mouse model with Leber congenital amaurosis phenotype. Molecular Vision. 24: 834-846. PMID 30713422  0.6
2018 Song C, Conlon TJ, Deng WT, Coleman KE, Zhu P, Plummer C, Mandapati S, Hoosear MV, Green KB, Sonnentag P, Sharma AK, Timmers A, Robinson P, Knop DR, Hauswirth WW, et al. Toxicology and Pharmacology of an AAV Vector Expressing Codon-optimized RPGR in RPGR-deficient Rd9 Mice. Human Gene Therapy. Clinical Development. PMID 30280954 DOI: 10.1089/humc.2018.168  0.48
2018 Bosco A, Anderson SR, Breen KT, Romero CO, Steele MR, Chiodo VA, Boye SL, Hauswirth WW, Tomlinson S, Vetter ML. Complement C3-Targeted Gene Therapy Restricts Onset and Progression of Neurodegeneration in Chronic Mouse Glaucoma. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 30217731 DOI: 10.1016/j.ymthe.2018.08.017  0.6
2018 Cideciyan AV, Sudharsan R, Dufour VL, Massengill MT, Iwabe S, Swider M, Lisi B, Sumaroka A, Marinho LF, Appelbaum T, Rossmiller B, Hauswirth WW, Jacobson SG, Lewin AS, Aguirre GD, et al. Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector. Proceedings of the National Academy of Sciences of the United States of America. PMID 30127005 DOI: 10.1073/pnas.1805055115  0.64
2018 Deng WT, Kolandaivelu S, Dinculescu A, Li J, Zhu P, Chiodo VA, Ramamurthy V, Hauswirth WW. Cone Phosphodiesterase-6γ' Subunit Augments Cone PDE6 Holoenzyme Assembly and Stability in a Mouse Model Lacking Both Rod and Cone PDE6 Catalytic Subunits. Frontiers in Molecular Neuroscience. 11: 233. PMID 30038560 DOI: 10.3389/fnmol.2018.00233  0.6
2018 Yang F, Ma H, Boye SL, Hauswirth WW, Ding XQ. Overexpression of Type 3 Iodothyronine Deiodinase Reduces Cone Death in the Leber Congenital Amaurosis Model Mice. Advances in Experimental Medicine and Biology. 1074: 125-131. PMID 29721936 DOI: 10.1007/978-3-319-75402-4_16  0.36
2018 Dinculescu A, Dyka FM, Min SH, Stupay RM, Hooper MJ, Smith WC, Hauswirth WW. Co-Expression of Wild-Type and Mutant S163R C1QTNF5 in Retinal Pigment Epithelium. Advances in Experimental Medicine and Biology. 1074: 61-66. PMID 29721928 DOI: 10.1007/978-3-319-75402-4_8  0.6
2018 Zhang Y, Deng WT, Du W, Zhu P, Li J, Xu F, Sun J, Gerstner CD, Baehr W, Boye SL, Zhao C, Hauswirth WW, Pang JJ. Publisher Correction: Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy. Scientific Reports. 8: 4807. PMID 29540812 DOI: 10.1038/s41598-018-23131-w  0.48
2018 Guziewicz KE, Cideciyan AV, Beltran WA, Komáromy AM, Dufour VL, Swider M, Iwabe S, Sumaroka A, Kendrick BT, Ruthel G, Chiodo VA, Héon E, Hauswirth WW, Jacobson SG, Aguirre GD. gene therapy corrects a diffuse retina-wide microdetachment modulated by light exposure. Proceedings of the National Academy of Sciences of the United States of America. PMID 29507198 DOI: 10.1073/pnas.1720662115  0.6
2018 Guy J, Feuer WJ, Davis JL, Porciatti V, Gonzalez PJ, Koilkonda RD, Yuan H, Hauswirth WW, Lam BL. Reply. Ophthalmology. 125: e15-e16. PMID 29389414 DOI: 10.1016/j.ophtha.2017.08.041  0.36
2018 Deng WT, Li J, Zhu P, Chiodo VA, Smith WC, Freedman B, Baehr W, Pang J, Hauswirth WW. Human L- and M-opsins restore M-cone function in a mouse model for human blue cone monochromacy. Molecular Vision. 24: 17-28. PMID 29386880  0.6
2018 Kady NM, Liu X, Lydic TA, Syed MH, Navitskaya S, Wang Q, Hammer SS, O'Reilly S, Huang C, Seregin SS, Amalfitano A, Chiodo VA, Boye SL, Hauswirth WW, Antonetti DA, et al. ELOVL4-Mediated Production of Very Long Chain Ceramides Stabilizes Tight Junctions and Prevents Diabetes-Induced Retinal Vascular Permeability. Diabetes. PMID 29362226 DOI: 10.2337/db17-1034  0.6
2017 Petersen-Jones SM, Occelli LM, Winkler PA, Lee W, Sparrow JR, Tsukikawa M, Boye SL, Chiodo V, Capasso JE, Becirovic E, Schön C, Seeliger MW, Levin AV, Michalakis S, Hauswirth WW, et al. Patients and animal models of CNGβ1-deficient retinitis pigmentosa support gene augmentation approach. The Journal of Clinical Investigation. PMID 29202463 DOI: 10.1172/JCI95161  0.36
2017 Ye GJ, Komaromy AM, Zeiss CJ, Calcedo R, Harman CD, Koehl KL, Stewart G, Iwabe S, Chiodo V, Hauswirth WW, Aguirre G, Chulay JD. Safety and Efficacy of AAV5 Vectors Expressing Human or Canine CNGB3 in CNGB3-mutant Dogs. Human Gene Therapy. Clinical Development. PMID 29020838 DOI: 10.1089/humc.2017.125  0.36
2017 Roddy GW, Yasumura D, Matthes MT, Alavi MV, Boye SL, Rosa RH, Fautsch MP, Hauswirth WW, LaVail MM. Long-term photoreceptor rescue in two rodent models of retinitis pigmentosa by adeno-associated virus delivery of Stanniocalcin-1. Experimental Eye Research. PMID 28974356 DOI: 10.1016/j.exer.2017.09.011  0.4
2017 Kanaan NM, Sellnow RC, Boye SL, Coberly B, Bennett A, Agbandje-McKenna M, Sortwell CE, Hauswirth WW, Boye SE, Manfredsson FP. Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS. Molecular Therapy. Nucleic Acids. 8: 184-197. PMID 28918020 DOI: 10.1016/j.omtn.2017.06.011  0.64
2017 Zhang Y, Deng WT, Du W, Zhu P, Li J, Xu F, Sun J, Gerstner CD, Baehr W, Sanford LB, Zhao C, Hauswirth WW, Pang JJ. Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy. Scientific Reports. 7: 6690. PMID 28751656 DOI: 10.1038/s41598-017-06982-7  0.48
2017 Mowat FM, Occelli LM, Bartoe JT, Gervais KJ, Bruewer AR, Querubin J, Dinculescu A, Boye SL, Hauswirth WW, Petersen-Jones SM. Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa. Frontiers in Neuroscience. 11: 342. PMID 28676737 DOI: 10.3389/fnins.2017.00342  0.48
2017 Guy J, Feuer WJ, Davis JL, Porciatti V, Gonzalez PJ, Koilkonda RD, Yuan H, Hauswirth WW, Lam BL. Gene Therapy for Leber Hereditary Optic Neuropathy: Low- and Medium-Dose Visual Results. Ophthalmology. PMID 28647203 DOI: 10.1016/j.ophtha.2017.05.016  0.36
2017 Beltran WA, Cideciyan AV, Boye SE, Ye GJ, Iwabe S, Dufour VL, Marinho LF, Swider M, Kosyk MS, Sha J, Boye SL, Peterson JJ, Witherspoon CD, Alexander JJ, Ying GS, ... ... Hauswirth WW, et al. Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 28566226 DOI: 10.1016/j.ymthe.2017.05.004  0.64
2017 Cabral-Miranda F, Nicoloso-Simões E, Adão-Novaes J, Chiodo V, Hauswirth WW, Linden R, Chiarini LB, Petrs-Silva H. rAAV8-733-Mediated Gene Transfer of CHIP/Stub-1 Prevents Hippocampal Neuronal Death in Experimental Brain Ischemia. Molecular Therapy : the Journal of the American Society of Gene Therapy. 25: 392-400. PMID 28153090 DOI: 10.1016/j.ymthe.2016.11.017  0.36
2017 Chen M, Maeng K, Nawab A, Francois RA, Bray JK, Reinhard MK, Boye SL, Hauswirth WW, Kaye FJ, Aslanidi GV, Srivastava A, Zajac-Kaye M. Efficient Gene Delivery and Expression in Pancreas and Pancreatic Tumors by Capsid-optimized AAV8 Vectors. Human Gene Therapy Methods. PMID 28125909 DOI: 10.1089/hgtb.2016.089  0.36
2016 Yang F, Ma H, Belcher J, Butler MR, Redmond TM, Boye SL, Hauswirth WW, Ding XQ. Targeting iodothyronine deiodinases locally in the retina is a therapeutic strategy for retinal degeneration. Faseb Journal : Official Publication of the Federation of American Societies For Experimental Biology. PMID 27623928 DOI: 10.1096/fj.201600715R  0.36
2016 Hickmott JW, Chen CY, Arenillas DJ, Korecki AJ, Lam SL, Molday LL, Bonaguro RJ, Zhou M, Chou AY, Mathelier A, Boye SL, Hauswirth WW, Molday RS, Wasserman WW, Simpson EM. PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina. Molecular Therapy. Methods & Clinical Development. 3: 16051. PMID 27556059 DOI: 10.1038/mtm.2016.51  0.36
2016 Ying G, Gerstner CD, Frederick JM, Boye SL, Hauswirth WW, Baehr W. Small GTPases Rab8a and Rab11a Are Dispensable for Rhodopsin Transport in Mouse Photoreceptors. Plos One. 11: e0161236. PMID 27529348 DOI: 10.1371/journal.pone.0161236  0.36
2016 Langlo CS, Patterson EJ, Higgins BP, Summerfelt P, Razeen MM, Erker LR, Parker M, Collison FT, Fishman GA, Kay CN, Zhang J, Weleber RG, Yang P, Wilson DJ, Pennesi ME, ... ... Hauswirth WW, et al. Residual Foveal Cone Structure in CNGB3-Associated Achromatopsia. Investigative Ophthalmology & Visual Science. 57: 3984-3995. PMID 27479814 DOI: 10.1167/iovs.16-19313  0.36
2016 Boye S, Alexander JJ, Witherspoon CD, Boye SL, Peterson JJ, Clark M, Sandefer KJ, Girkin CA, Hauswirth WW, Gamlin PD. Highly Efficient Delivery of AAV vectors to the Primate Retina. Human Gene Therapy. PMID 27439313 DOI: 10.1089/hum.2016.085  0.36
2016 Liu X, Dreffs A, Díaz-Coránguez M, Runkle EA, Gardner TW, Chiodo VA, Hauswirth WW, Antonetti DA. Occludin S490 Phosphorylation Regulates Vascular Endothelial Growth Factor-Induced Retinal Neovascularization. The American Journal of Pathology. PMID 27423695 DOI: 10.1016/j.ajpath.2016.04.018  0.6
2016 Weleber RG, Pennesi ME, Wilson DJ, Kaushal S, Erker LR, Jensen L, McBride MT, Flotte TR, Humphries M, Calcedo R, Hauswirth WW, Chulay JD, Stout JT. Results at 2 Years after Gene Therapy for RPE65-Deficient Leber Congenital Amaurosis and Severe Early-Childhood-Onset Retinal Dystrophy. Ophthalmology. PMID 27102010 DOI: 10.1016/j.ophtha.2016.03.003  0.36
2016 Boyd RF, Sledge DG, Boye SL, Boye SE, Hauswirth WW, Komáromy AM, Petersen-Jones SM, Bartoe JT. Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs. Gene Therapy. 23: 400. PMID 27052928 DOI: 10.1038/gt.2016.10  0.64
2016 Boyd RF, Boye SL, Conlon TJ, Erger KE, Sledge DG, Langohr IM, Hauswirth WW, Komáromy AM, Boye SE, Petersen-Jones SM, Bartoe JT. Reduced retinal transduction and enhanced transgene-directed immunogenicity with intravitreal delivery of rAAV following posterior vitrectomy in dogs. Gene Therapy. PMID 27052802 DOI: 10.1038/gt.2016.31  0.36
2016 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. Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia. Human Gene Therapy. Clinical Development. 27: 37-48. PMID 27003753 DOI: 10.1089/humc.2015.164  0.36
2016 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. Safety and Biodistribution Evaluation in CNGB3-Deficient Mice of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia. Human Gene Therapy. Clinical Development. 27: 27-36. PMID 27003752 DOI: 10.1089/humc.2015.163  0.36
2016 Yan B, Vakulenko M, Min SH, Hauswirth WW, Nirenberg S. Maintaining ocular safety with light exposure, focusing on devices for optogenetic stimulation. Vision Research. PMID 26882975 DOI: 10.1016/j.visres.2016.01.006  0.36
2016 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, et al. AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy. Plos One. 11: e0148874. PMID 26881841 DOI: 10.1371/journal.pone.0148874  0.36
2016 Ildefonso CJ, Jaime H, Brown EE, Iwata RL, Ahmed CM, Massengill MT, Biswal MR, Boye SE, Hauswirth WW, Ash JD, Li Q, Lewin AS. Targeting the Nrf2 Signaling Pathway in the Retina With a Gene-Delivered Secretable and Cell-Penetrating Peptide. Investigative Ophthalmology & Visual Science. 57: 372-86. PMID 26842755 DOI: 10.1167/iovs.15-17703  0.36
2016 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, ... ... Hauswirth W, et al. Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial. Human Genetics. PMID 26825853 DOI: 10.1007/s00439-016-1637-y  0.36
2016 LaVail MM, Yasumura D, Matthes MT, Yang H, Hauswirth WW, Deng WT, Vollrath D. Gene Therapy for MERTK-Associated Retinal Degenerations. Advances in Experimental Medicine and Biology. 854: 487-93. PMID 26427450 DOI: 10.1007/978-3-319-17121-0_65  1
2015 Berns KI, Byrne BJ, Flotte TR, Gao G, Hauswirth WW, Herzog RW, Muzyczka N, VandenDriessche T, Xiao X, Zolotukhin S, Srivastava A. Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma? Human Gene Therapy. 26: 779-81. PMID 26690810 DOI: 10.1089/hum.2015.29014.kib  1
2015 Ye GJ, Budzynski E, Sonnentag P, Nork TM, Sheibani N, Gurel Z, Boye SL, Peterson JJ, Boye SE, Hauswirth WW, Chulay JD. Cone-specific promoters for gene therapy of achromatopsia and other retinal diseases. Human Gene Therapy. PMID 26603570 DOI: 10.1089/hum.2015.130  0.36
2015 Dinculescu A, Min SH, Dyka FM, Deng WT, Stupay RM, Chiodo V, Smith WC, Hauswirth WW. Pathological Effects of Mutant C1QTNF5 (S163R) Expression in Murine Retinal Pigment Epithelium. Investigative Ophthalmology & Visual Science. 56: 6971-80. PMID 26513502 DOI: 10.1167/iovs.15-17166  0.36
2015 Boyd RF, Sledge DG, Boye SL, Boye SE, Hauswirth WW, Komáromy AM, Petersen-Jones SM, Bartoe JT. Photoreceptor-targeted gene delivery using intravitreally administered AAV vectors in dogs. Gene Therapy. PMID 26467396 DOI: 10.1038/gt.2015.96  0.36
2015 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. Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease. Proceedings of the National Academy of Sciences of the United States of America. 112: E5844-53. PMID 26460017 DOI: 10.1073/pnas.1509914112  0.36
2015 Yu H, Koilkonda RD, Chou TH, Porciatti V, Mehta A, Hentall ID, Chiodo VA, Boye SL, Hauswirth WW, Lewin AS, Guy J. Consequences of zygote injection and germline transfer of mutant human mitochondrial DNA in mice. Proceedings of the National Academy of Sciences of the United States of America. PMID 26438859 DOI: 10.1073/pnas.1506129112  0.36
2015 Lipinski DM, Reid CA, Boye SL, Peterson JJ, Qi X, Boye SE, Boulton ME, Hauswirth WW. Systemic Vascular Transduction by Capsid Mutant Adeno-Associated Virus After Intravenous Injection. Human Gene Therapy. PMID 26359319 DOI: 10.1089/hum.2015.097  0.36
2015 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. Gene Therapy Fully Restores Vision to the All-Cone Nrl(-/-)Gucy2e(-/-) Mouse Model of Leber Congenital Amaurosis-1. Human Gene Therapy. 26: 575-92. PMID 26247368 DOI: 10.1089/hum.2015.053  0.36
2015 Jacobson SG, Cideciyan AV, Aguirre GD, Roman AJ, Sumaroka A, Hauswirth WW, Palczewski K. Improvement in vision: a new goal for treatment of hereditary retinal degenerations. Expert Opinion On Orphan Drugs. 3: 563-575. PMID 26246977 DOI: 10.1517/21678707.2015.1030393  1
2015 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. Gene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 Achromatopsia. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 26087757 DOI: 10.1038/mt.2015.114  1
2015 Deng WT, Dyka FM, Dinculescu A, Li J, Zhu P, Chiodo V, Boye SL, Conlon TJ, Erger KE, Cossette T, Hauswirth WW. Stability and Safety of an AAV Vector for Treating RPGR-ORF15 X-linked Retinitis Pigmentosa. Human Gene Therapy. PMID 26076799 DOI: 10.1089/hum.2015.035  0.36
2015 Bogner B, Boye SL, Min SH, Peterson JJ, Ruan Q, Zhang Z, Reitsamer HA, Hauswirth WW, Boye SE. Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and Rat. Plos One. 10: e0128759. PMID 26052939 DOI: 10.1371/journal.pone.0128759  1
2015 Zhong H, Eblimit A, Moayedi Y, Boye SL, Chiodo VA, Chen Y, Li Y, Nichols RM, Hauswirth WW, Chen R, Mardon G. AAV8(Y733F)-mediated gene therapy in a Spata7 knockout mouse model of Leber congenital amaurosis and retinitis pigmentosa. Gene Therapy. PMID 25965394 DOI: 10.1038/gt.2015.42  1
2015 Jacobson SG, Cideciyan AV, Roman AJ, Sumaroka A, Schwartz SB, Heon E, Hauswirth WW. Improvement and decline in vision with gene therapy in childhood blindness. The New England Journal of Medicine. 372: 1920-6. PMID 25936984 DOI: 10.1056/NEJMoa1412965  1
2015 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, ... ... Hauswirth WW, et al. Vitreal delivery of AAV vectored Cnga3 restores cone function in CNGA3-/-/Nrl-/- mice, an all-cone model of CNGA3 achromatopsia†. Human Molecular Genetics. 24: 3699-707. PMID 25855802 DOI: 10.1093/hmg/ddv114  1
2015 Ildefonso CJ, Jaime H, Biswal MR, Boye SE, Li Q, Hauswirth WW, Lewin AS. Gene therapy with the caspase activation and recruitment domain reduces the ocular inflammatory response. Molecular Therapy : the Journal of the American Society of Gene Therapy. 23: 875-84. PMID 25698151 DOI: 10.1038/mt.2015.30  1
2015 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, et al. Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano Symposium. Investigative Ophthalmology & Visual Science. 56: 918-31. PMID 25667399 DOI: 10.1167/iovs.14-16049  1
2015 Talla V, Koilkonda R, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Guy J. Complex I subunit gene therapy with NDUFA6 ameliorates neurodegeneration in EAE. Investigative Ophthalmology & Visual Science. 56: 1129-40. PMID 25613946 DOI: 10.1167/iovs.14-15950  1
2015 Benskey MJ, Kuhn NC, Galligan JJ, Garcia J, Boye SE, Hauswirth WW, Mueller C, Boye SL, Manfredsson FP. Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants. Molecular Therapy : the Journal of the American Society of Gene Therapy. 23: 488-500. PMID 25592336 DOI: 10.1038/mt.2015.7  1
2015 Cideciyan AV, Aguirre GK, Jacobson SG, Butt OH, Schwartz SB, Swider M, Roman AJ, Sadigh S, Hauswirth WW. Pseudo-fovea formation after gene therapy for RPE65-LCA. Investigative Ophthalmology & Visual Science. 56: 526-37. PMID 25537204 DOI: 10.1167/iovs.14-15895  1
2015 Kwong JM, Gu L, Nassiri N, Bekerman V, Kumar-Singh R, Rhee KD, Yang XJ, Hauswirth WW, Caprioli J, Piri N. AAV-mediated and pharmacological induction of Hsp70 expression stimulates survival of retinal ganglion cells following axonal injury. Gene Therapy. 22: 138-45. PMID 25427613 DOI: 10.1038/gt.2014.105  1
2015 Ildefonso CJ, Jaime H, Rahman MM, Li Q, Boye SE, Hauswirth WW, Lucas AR, McFadden G, Lewin AS. Gene delivery of a viral anti-inflammatory protein to combat ocular inflammation. Human Gene Therapy. 26: 59-68. PMID 25420215 DOI: 10.1089/hum.2014.089  1
2015 Beltran WA, Cideciyan AV, Lewin AS, Hauswirth WW, Jacobson SG, Aguirre GD. Gene augmentation for X-linked retinitis pigmentosa caused by mutations in RPGR. Cold Spring Harbor Perspectives in Medicine. 5: a017392. PMID 25301933 DOI: 10.1101/cshperspect.a017392  1
2015 Ku CA, Chiodo VA, Boye SL, Hayes A, Goldberg AF, Hauswirth WW, Ramamurthy V. Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model. Human Molecular Genetics. 24: 670-84. PMID 25274777 DOI: 10.1093/hmg/ddu487  1
2015 Robinson PM, Kim SS, Sriram S, Tuli S, Hauswirth WW, Lewin AS, Schultz GS. Reduction of Profibrotic Connective Tissue Growth Factor in Wounded Rat Corneas Using a Ribozyme Expressed in Self Complimentary Adeno-Associated Virus Wound Healing: Cellular Mechanisms, Alternative Therapies and Clinical Outcomes. 25-42.  0.64
2014 Cabral Miranda F, Adão-Novaes J, Hauswirth WW, Linden R, Petrs-Silva H, Chiarini LB. CHIP, a carboxy terminus HSP-70 interacting protein, prevents cell death induced by endoplasmic reticulum stress in the central nervous system. Frontiers in Cellular Neuroscience. 8: 438. PMID 25620910 DOI: 10.3389/fncel.2014.00438  1
2014 Jiang K, Wright KL, Zhu P, Szego MJ, Bramall AN, Hauswirth WW, Li Q, Egan SE, McInnes RR. STAT3 promotes survival of mutant photoreceptors in inherited photoreceptor degeneration models. Proceedings of the National Academy of Sciences of the United States of America. 111: E5716-23. PMID 25512545 DOI: 10.1073/pnas.1411248112  1
2014 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, et al. DICER1/Alu RNA dysmetabolism induces Caspase-8-mediated cell death in age-related macular degeneration. Proceedings of the National Academy of Sciences of the United States of America. 111: 16082-7. PMID 25349431 DOI: 10.1073/pnas.1403814111  1
2014 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. LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile. Investigative Ophthalmology & Visual Science. 55: 7739-53. PMID 25342621 DOI: 10.1167/iovs.14-15388  1
2014 Ramamurthy V, Jolicoeur C, Koutroumbas D, Mühlhans J, Le YZ, Hauswirth WW, Giessl A, Cayouette M. Numb regulates the polarized delivery of cyclic nucleotide-gated ion channels in rod photoreceptor cilia. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 34: 13976-87. PMID 25319694 DOI: 10.1523/JNEUROSCI.1938-14.2014  1
2014 Regus-Leidig H, Fuchs M, Löhner M, Leist SR, Leal-Ortiz S, Chiodo VA, Hauswirth WW, Garner CC, Brandstätter JH. In vivo knockdown of Piccolino disrupts presynaptic ribbon morphology in mouse photoreceptor synapses. Frontiers in Cellular Neuroscience. 8: 259. PMID 25232303 DOI: 10.3389/fncel.2014.00259  1
2014 Ezra-Elia R, Banin E, Honig H, Rosov A, Obolensky A, Averbukh E, Hauswirth WW, Gootwine E, Ofri R. Flicker cone function in normal and day blind sheep: a large animal model for human achromatopsia caused by CNGA3 mutation. Documenta Ophthalmologica. Advances in Ophthalmology. 129: 141-50. PMID 25204753 DOI: 10.1007/s10633-014-9458-6  1
2014 Hauswirth WW. Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus. Human Gene Therapy. 25: 671-8. PMID 25136913 DOI: 10.1089/hum.2014.2530  1
2014 Talla V, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Guy J. Gene therapy with mitochondrial heat shock protein 70 suppresses visual loss and optic atrophy in experimental autoimmune encephalomyelitis. Investigative Ophthalmology & Visual Science. 55: 5214-26. PMID 25015358 DOI: 10.1167/iovs.14-14688  1
2014 Zhu Y, Xu J, Hauswirth WW, DeVries SH. Genetically targeted binary labeling of retinal neurons. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 34: 7845-61. PMID 24899708 DOI: 10.1523/JNEUROSCI.2960-13.2014  1
2014 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, ... ... Hauswirth WW, et al. Targeted CNS Delivery Using Human MiniPromoters and Demonstrated Compatibility with Adeno-Associated Viral Vectors. Molecular Therapy. Methods & Clinical Development. 1: 5. PMID 24761428 DOI: 10.1038/mtm.2013.5  0.64
2014 Wilson AM, Chiodo VA, Boye SL, Brecha NC, Hauswirth WW, Di Polo A. Inhibitor of apoptosis-stimulating protein of p53 (iASPP) is required for neuronal survival after axonal injury. Plos One. 9: e94175. PMID 24714389 DOI: 10.1371/journal.pone.0094175  1
2014 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. Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy. Plos One. 9: e92928. PMID 24671090 DOI: 10.1371/journal.pone.0092928  1
2014 Dinculescu A, Min SH, Deng WT, Li Q, Hauswirth WW. Gene therapy in the rd6 mouse model of retinal degeneration. Advances in Experimental Medicine and Biology. 801: 711-8. PMID 24664762 DOI: 10.1007/978-1-4614-3209-8_89  1
2014 Dyka FM, Boye SL, Ryals RC, Chiodo VA, Boye SE, Hauswirth WW. Cone specific promoter for use in gene therapy of retinal degenerative diseases. Advances in Experimental Medicine and Biology. 801: 695-701. PMID 24664760 DOI: 10.1007/978-1-4614-3209-8_87  1
2014 Dyka FM, Boye SL, Chiodo VA, Hauswirth WW, Boye SE. Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A. Human Gene Therapy Methods. 25: 166-77. PMID 24568220 DOI: 10.1089/hgtb.2013.212  1
2014 Dai X, Han J, Qi Y, Zhang H, Xiang L, Lv J, Li J, Deng WT, Chang B, Hauswirth WW, Pang JJ. AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice. Investigative Ophthalmology & Visual Science. 55: 1724-34. PMID 24557352 DOI: 10.1167/iovs.13-13654  1
2014 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. Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial. Jama Ophthalmology. 132: 409-20. PMID 24457989 DOI: 10.1001/jamaophthalmol.2013.7630  1
2014 Mowat FM, Gornik KR, Dinculescu A, Boye SL, Hauswirth WW, Petersen-Jones SM, Bartoe JT. Tyrosine capsid-mutant AAV vectors for gene delivery to the canine retina from a subretinal or intravitreal approach. Gene Therapy. 21: 96-105. PMID 24225638 DOI: 10.1038/gt.2013.64  1
2014 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. Reprogramming adipose tissue-derived mesenchymal stem cells into pluripotent stem cells by a mutant adeno-associated viral vector Human Gene Therapy Methods. 25: 72-82. PMID 24191859 DOI: 10.1089/hgtb.2013.011  1
2013 Guziewicz KE, Zangerl B, Komáromy AM, Iwabe S, Chiodo VA, Boye SL, Hauswirth WW, Beltran WA, Aguirre GD. Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects. Plos One. 8: e75666. PMID 24143172 DOI: 10.1371/journal.pone.0075666  1
2013 Chavala SH, Kim Y, Tudisco L, Cicatiello V, Milde T, Kerur N, Claros N, Yanni S, Guaiquil VH, Hauswirth WW, Penn JS, Rafii S, De Falco S, Lee TC, Ambati J. Retinal angiogenesis suppression through small molecule activation of p53. The Journal of Clinical Investigation. 123: 4170-81. PMID 24018558 DOI: 10.1172/JCI67315  1
2013 Yu H, Mehta A, Wang G, Hauswirth WW, Chiodo V, Boye SL, Guy J. Next-generation sequencing of mitochondrial targeted AAV transfer of human ND4 in mice. Molecular Vision. 19: 1482-91. PMID 23869167  1
2013 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. Cone phosphodiesterase-6α' restores rod function and confers distinct physiological properties in the rod phosphodiesterase-6β-deficient rd10 mouse. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 33: 11745-53. PMID 23864662 DOI: 10.1523/JNEUROSCI.1536-13.2013  1
2013 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, ... ... Hauswirth WW, et al. Photoreceptor avascular privilege is shielded by soluble VEGF receptor-1. Elife. 2: e00324. PMID 23795287 DOI: 10.7554/eLife.00324  1
2013 Cideciyan AV, Jacobson SG, Beltran WA, Hauswirth WW, Aguirre GD. Reply to Townes-Anderson: RPE65 gene therapy does not alter the natural history of retinal degeneration. Proceedings of the National Academy of Sciences of the United States of America. 110: E1706. PMID 23789127 DOI: 10.1073/pnas.1304296110  1
2013 Talla V, Yu H, Chou TH, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Lewin AS, Guy J. NADH-dehydrogenase type-2 suppresses irreversible visual loss and neurodegeneration in the EAE animal model of MS. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 1876-88. PMID 23752309 DOI: 10.1038/mt.2013.104  1
2013 Molday LL, Djajadi H, Yan P, Szczygiel L, Boye SL, Chiodo VA, Gregory-Evans K, Sarunic MV, Hauswirth WW, Molday RS. RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12. Human Molecular Genetics. 22: 3894-905. PMID 23740938 DOI: 10.1093/hmg/ddt244  1
2013 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, et al. Preclinical potency and safety studies of an AAV2-mediated gene therapy vector for the treatment of MERTK associated retinitis pigmentosa. Human Gene Therapy. Clinical Development. 24: 23-8. PMID 23692380 DOI: 10.1089/humc.2013.037  1
2013 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. Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors. Plos One. 8: e62097. PMID 23637972 DOI: 10.1371/journal.pone.0062097  1
2013 Komáromy AM, Rowlan JS, Corr AT, Reinstein SL, Boye SL, Cooper AE, Gonzalez A, Levy B, Wen R, Hauswirth WW, Beltran WA, Aguirre GD. Transient photoreceptor deconstruction by CNTF enhances rAAV-mediated cone functional rescue in late stage CNGB3-achromatopsia. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 1131-41. PMID 23568263 DOI: 10.1038/mt.2013.50  1
2013 Bruewer AR, Mowat FM, Bartoe JT, Boye SL, Hauswirth WW, Petersen-Jones SM. Evaluation of lateral spread of transgene expression following subretinal AAV-mediated gene delivery in dogs. Plos One. 8: e60218. PMID 23560080 DOI: 10.1371/journal.pone.0060218  1
2013 Jiang L, Li TZ, Boye SE, Hauswirth WW, Frederick JM, Baehr W. RNAi-mediated gene suppression in a GCAP1(L151F) cone-rod dystrophy mouse model. Plos One. 8: e57676. PMID 23472098 DOI: 10.1371/journal.pone.0057676  1
2013 Bramall AN, Szego MJ, Pacione LR, Chang I, Diez E, D'Orleans-Juste P, Stewart DJ, Hauswirth WW, Yanagisawa M, McInnes RR. Endothelin-2-mediated protection of mutant photoreceptors in inherited photoreceptor degeneration. Plos One. 8: e58023. PMID 23469133 DOI: 10.1371/journal.pone.0058023  1
2013 Welsbie DS, Yang Z, Ge Y, Mitchell KL, Zhou X, Martin SE, Berlinicke CA, Hackler L, Fuller J, Fu J, Cao LH, Han B, Auld D, Xue T, Hirai S, ... ... Hauswirth WW, et al. Functional genomic screening identifies dual leucine zipper kinase as a key mediator of retinal ganglion cell death. Proceedings of the National Academy of Sciences of the United States of America. 110: 4045-50. PMID 23431148 DOI: 10.1073/pnas.1211284110  1
2013 Boye SE, Boye SL, Lewin AS, Hauswirth WW. A comprehensive review of retinal gene therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 509-19. PMID 23358189 DOI: 10.1038/mt.2012.280  1
2013 Lopes VS, Boye SE, Louie CM, Boye S, Dyka F, Chiodo V, Fofo H, Hauswirth WW, Williams DS. Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus. Gene Therapy. 20: 824-33. PMID 23344065 DOI: 10.1038/gt.2013.3  1
2013 Weitz AC, Behrend MR, Lee NS, Klein RL, Chiodo VA, Hauswirth WW, Humayun MS, Weiland JD, Chow RH. Imaging the response of the retina to electrical stimulation with genetically encoded calcium indicators. Journal of Neurophysiology. 109: 1979-88. PMID 23343890 DOI: 10.1152/jn.00852.2012  1
2013 Cideciyan AV, Jacobson SG, Beltran WA, Sumaroka A, Swider M, Iwabe S, Roman AJ, Olivares MB, Schwartz SB, Komáromy AM, Hauswirth WW, Aguirre GD. Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement. Proceedings of the National Academy of Sciences of the United States of America. 110: E517-25. PMID 23341635 DOI: 10.1073/pnas.1218933110  1
2013 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, et al. AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis. Human Gene Therapy. 24: 189-202. PMID 23210611 DOI: 10.1089/hum.2012.193  1
2013 Ruan Q, Xi L, Boye SL, Han S, Chen ZJ, Hauswirth WW, Lewin AS, Boulton ME, Law BK, Jiang WG, Jiang H, Cai J. Development of an anti-angiogenic therapeutic model combining scAAV2-delivered siRNAs and noninvasive photoacoustic imaging of tumor vasculature development. Cancer Letters. 332: 120-9. PMID 23196055 DOI: 10.1016/j.canlet.2012.11.016  1
2013 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. scAAV-mediated gene transfer of interleukin-1-receptor antagonist to synovium and articular cartilage in large mammalian joints. Gene Therapy. 20: 670-7. PMID 23151520 DOI: 10.1038/gt.2012.81  1
2012 Bermingham-McDonogh O, Corwin JT, Hauswirth WW, Heller S, Reed R, Reh TA. Regenerative medicine for the special senses: restoring the inputs. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 32: 14053-7. PMID 23055472 DOI: 10.1523/JNEUROSCI.3336-12.2012  1
2012 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, et al. ERK1/2 activation is a therapeutic target in age-related macular degeneration. Proceedings of the National Academy of Sciences of the United States of America. 109: 13781-6. PMID 22869729 DOI: 10.1073/pnas.1206494109  1
2012 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. The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina. Human Gene Therapy. 23: 1101-15. PMID 22845794 DOI: 10.1089/hum.2012.125  1
2012 Light AC, Zhu Y, Shi J, Saszik S, Lindstrom S, Davidson L, Li X, Chiodo VA, Hauswirth WW, Li W, DeVries SH. Organizational motifs for ground squirrel cone bipolar cells. The Journal of Comparative Neurology. 520: 2864-87. PMID 22778006 DOI: 10.1002/cne.23068  1
2012 Yu H, Ozdemir SS, Koilkonda RD, Chou TH, Porciatti V, Chiodo V, Boye SL, Hauswirth WW, Lewin AS, Guy J. Mutant NADH dehydrogenase subunit 4 gene delivery to mitochondria by targeting sequence-modified adeno-associated virus induces visual loss and optic atrophy in mice. Molecular Vision. 18: 1668-83. PMID 22773905  1
2012 Yao J, Jia L, Khan N, Zheng QD, Moncrief A, Hauswirth WW, Thompson DA, Zacks DN. Caspase inhibition with XIAP as an adjunct to AAV vector gene-replacement therapy: improving efficacy and prolonging the treatment window. Plos One. 7: e37197. PMID 22615940 DOI: 10.1371/journal.pone.0037197  1
2012 Pi L, Shenoy AK, Liu J, Kim S, Nelson N, Xia H, Hauswirth WW, Petersen BE, Schultz GS, Scott EW. CCN2/CTGF regulates neovessel formation via targeting structurally conserved cystine knot motifs in multiple angiogenic regulators. Faseb Journal : Official Publication of the Federation of American Societies For Experimental Biology. 26: 3365-79. PMID 22611085 DOI: 10.1096/fj.11-200154  1
2012 Kunte MM, Choudhury S, Manheim JF, Shinde VM, Miura M, Chiodo VA, Hauswirth WW, Gorbatyuk OS, Gorbatyuk MS. ER stress is involved in T17M rhodopsin-induced retinal degeneration. Investigative Ophthalmology & Visual Science. 53: 3792-800. PMID 22589437 DOI: 10.1167/iovs.11-9235  1
2012 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, et al. DICER1 loss and Alu RNA induce age-related macular degeneration via the NLRP3 inflammasome and MyD88. Cell. 149: 847-59. PMID 22541070 DOI: 10.1016/j.cell.2012.03.036  1
2012 Yu H, Koilkonda RD, Chou TH, Porciatti V, Ozdemir SS, Chiodo V, Boye SL, Boye SE, Hauswirth WW, Lewin AS, Guy J. Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model. Proceedings of the National Academy of Sciences of the United States of America. 109: E1238-47. PMID 22523243 DOI: 10.1073/pnas.1119577109  1
2012 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, ... ... Hauswirth WW, et al. AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia. Plos One. 7: e35250. PMID 22509403 DOI: 10.1371/journal.pone.0035250  1
2012 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, et al. Tyrosine-mutant AAV8 delivery of human MERTK provides long-term retinal preservation in RCS rats. Investigative Ophthalmology & Visual Science. 53: 1895-904. PMID 22408006 DOI: 10.1167/iovs.11-8831  1
2012 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, et al. Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. Proceedings of the National Academy of Sciences of the United States of America. 109: 2132-7. PMID 22308428 DOI: 10.1073/pnas.1118847109  1
2012 Mao H, Gorbatyuk MS, Rossmiller B, Hauswirth WW, Lewin AS. Long-term rescue of retinal structure and function by rhodopsin RNA replacement with a single adeno-associated viral vector in P23H RHO transgenic mice. Human Gene Therapy. 23: 356-66. PMID 22289036 DOI: 10.1089/hum.2011.213  1
2012 Qi X, Cai J, Ruan Q, Liu L, Boye SL, Chen Z, Hauswirth WW, Ryals RC, Shaw L, Caballero S, Grant MB, Boulton ME. γ-Secretase inhibition of murine choroidal neovascularization is associated with reduction of superoxide and proinflammatory cytokines. Investigative Ophthalmology & Visual Science. 53: 574-85. PMID 22205609 DOI: 10.1167/iovs.11-8728  1
2012 Petrs-Silva H, Yasumura D, Matthes MT, LaVail MM, Lewin AS, Hauswirth WW. Suppression of rds expression by siRNA and gene replacement strategies for gene therapy using rAAV vector. Advances in Experimental Medicine and Biology. 723: 215-23. PMID 22183336 DOI: 10.1007/978-1-4614-0631-0_29  1
2012 Mao H, Gorbatyuk MS, Hauswirth WW, Lewin AS. Gene delivery of wild-type rhodopsin rescues retinal function in an autosomal dominant retinitis pigmentosa mouse model. Advances in Experimental Medicine and Biology. 723: 199-205. PMID 22183334 DOI: 10.1007/978-1-4614-0631-0_27  1
2012 Gorbatyuk MS, Gorbatyuk OS, LaVail MM, Lin JH, Hauswirth WW, Lewin AS. Functional rescue of P23H rhodopsin photoreceptors by gene delivery. Advances in Experimental Medicine and Biology. 723: 191-7. PMID 22183333 DOI: 10.1007/978-1-4614-0631-0_26  1
2012 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, ... ... Hauswirth WW, et al. Gene therapy for retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of concept. Human Gene Therapy. 23: 367-76. PMID 22142163 DOI: 10.1089/hum.2011.169  1
2012 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, ... ... Hauswirth WW, et al. Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Archives of Ophthalmology (Chicago, Ill. : 1960). 130: 9-24. PMID 21911650 DOI: 10.1001/archophthalmol.2011.298  1
2012 Verma A, Shan Z, Lei B, Yuan L, Liu X, Nakagawa T, Grant MB, Lewin AS, Hauswirth WW, Raizada MK, Li Q. ACE2 and Ang-(1-7) confer protection against development of diabetic retinopathy. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 28-36. PMID 21792177 DOI: 10.1038/mt.2011.155  1
2012 Wang H, Murphy R, Taaffe D, Yin S, Xia L, Hauswirth WW, Bance M, Robertson GS, Wang J. Efficient cochlear gene transfection in guinea-pigs with adeno-associated viral vectors by partial digestion of round window membrane. Gene Therapy. 19: 255-63. PMID 21697953 DOI: 10.1038/gt.2011.91  1
2012 Tuo J, Pang JJ, Cao X, Shen D, Zhang J, Scaria A, Wadsworth SC, Pechan P, Boye SL, Hauswirth WW, Chan CC. AAV5-mediated sFLT01 gene therapy arrests retinal lesions in Ccl2(-/-)/Cx3cr1(-/-) mice. Neurobiology of Aging. 33: 433.e1-10. PMID 21397984 DOI: 10.1016/j.neurobiolaging.2011.01.009  1
2011 Doroudchi MM, Greenberg KP, Zorzos AN, Hauswirth WW, Fonstad CG, Horsager A, Boyden ES. Towards optogenetic sensory replacement. Conference Proceedings : ... Annual International Conference of the Ieee Engineering in Medicine and Biology Society. Ieee Engineering in Medicine and Biology Society. Annual Conference. 2011: 3139-41. PMID 22255005 DOI: 10.1109/IEMBS.2011.6090856  1
2011 Jiang L, Zhang H, Dizhoor AM, Boye SE, Hauswirth WW, Frederick JM, Baehr W. Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model. Proceedings of the National Academy of Sciences of the United States of America. 108: 18476-81. PMID 22042849 DOI: 10.1073/pnas.1112758108  1
2011 Cai J, Chen Z, Ruan Q, Han S, Liu L, Qi X, Boye SL, Hauswirth WW, Grant MB, Boulton ME. γ-Secretase and presenilin mediate cleavage and phosphorylation of vascular endothelial growth factor receptor-1. The Journal of Biological Chemistry. 286: 42514-23. PMID 22016384 DOI: 10.1074/jbc.M111.296590  1
2011 Pi L, Xia H, Liu J, Shenoy AK, Hauswirth WW, Scott EW. Role of connective tissue growth factor in the retinal vasculature during development and ischemia. Investigative Ophthalmology & Visual Science. 52: 8701-10. PMID 21969300 DOI: 10.1167/iovs.11-7870  1
2011 Ku CA, Chiodo VA, Boye SL, Goldberg AF, Li T, Hauswirth WW, Ramamurthy V. Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis. Human Molecular Genetics. 20: 4569-81. PMID 21880665 DOI: 10.1093/hmg/ddr391  1
2011 Boye SL, Conlon T, Erger K, Ryals R, Neeley A, Cossette T, Pang J, Dyka FM, Hauswirth WW, Boye SE. Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse. Investigative Ophthalmology & Visual Science. 52: 7098-108. PMID 21778276 DOI: 10.1167/iovs.11-7867  1
2011 Ryals RC, Boye SL, Dinculescu A, Hauswirth WW, Boye SE. Quantifying transduction efficiencies of unmodified and tyrosine capsid mutant AAV vectors in vitro using two ocular cell lines. Molecular Vision. 17: 1090-102. PMID 21552473  1
2011 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. Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 1220-9. PMID 21505421 DOI: 10.1038/mt.2011.69  1
2011 Chan F, Hauswirth WW, Wensel TG, Wilson JH. Efficient mutagenesis of the rhodopsin gene in rod photoreceptor neurons in mice. Nucleic Acids Research. 39: 5955-66. PMID 21478169 DOI: 10.1093/nar/gkr196  1
2011 Simons DL, Boye SL, Hauswirth WW, Wu SM. Gene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model. Proceedings of the National Academy of Sciences of the United States of America. 108: 6276-81. PMID 21444805 DOI: 10.1073/pnas.1019222108  1
2011 Peden MC, Min J, Meyers C, Lukowski Z, Li Q, Boye SL, Levine M, Hauswirth WW, Ratnakaram R, Dawson W, Smith WC, Sherwood MB. Ab-externo AAV-mediated gene delivery to the suprachoroidal space using a 250 micron flexible microcatheter. Plos One. 6: e17140. PMID 21347253 DOI: 10.1371/journal.pone.0017140  1
2011 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, et al. DICER1 deficit induces Alu RNA toxicity in age-related macular degeneration. Nature. 471: 325-30. PMID 21297615 DOI: 10.1038/nature09830  1
2011 Zou J, Luo L, Shen Z, Chiodo VA, Ambati BK, Hauswirth WW, Yang J. Whirlin replacement restores the formation of the USH2 protein complex in whirlin knockout photoreceptors. Investigative Ophthalmology & Visual Science. 52: 2343-51. PMID 21212183 DOI: 10.1167/iovs.10-6141  1
2011 Li X, Li W, Dai X, Kong F, Zheng Q, Zhou X, Lü F, Chang B, Rohrer B, Hauswirth WW, Qu J, Pang JJ. Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis. Investigative Ophthalmology & Visual Science. 52: 7-15. PMID 21169527 DOI: 10.1167/iovs.10-6138  1
2011 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. Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 234-42. PMID 21139570 DOI: 10.1038/mt.2010.273  1
2011 Mao H, James T, Schwein A, Shabashvili AE, Hauswirth WW, Gorbatyuk MS, Lewin AS. AAV delivery of wild-type rhodopsin preserves retinal function in a mouse model of autosomal dominant retinitis pigmentosa. Human Gene Therapy. 22: 567-75. PMID 21126223 DOI: 10.1089/hum.2010.140  1
2011 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, ... ... Hauswirth WW, et al. Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 293-301. PMID 21045809 DOI: 10.1038/mt.2010.234  1
2011 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. Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 260-5. PMID 20978476 DOI: 10.1038/mt.2010.230  1
2011 Yao J, Feathers KL, Khanna H, Thompson D, Tsilfidis C, Hauswirth WW, Heckenlively JR, Swaroop A, Zacks DN. XIAP therapy increases survival of transplanted rod precursors in a degenerating host retina. Investigative Ophthalmology & Visual Science. 52: 1567-72. PMID 20926819 DOI: 10.1167/iovs.10-5998  1
2011 Västinsalo H, Jalkanen R, Dinculescu A, Isosomppi J, Geller S, Flannery JG, Hauswirth WW, Sankila EM. Alternative splice variants of the USH3A gene Clarin 1 (CLRN1). European Journal of Human Genetics : Ejhg. 19: 30-5. PMID 20717163 DOI: 10.1038/ejhg.2010.140  1
2010 Lam BL, Feuer WJ, Abukhalil F, Porciatti V, Hauswirth WW, Guy J. Leber hereditary optic neuropathy gene therapy clinical trial recruitment: year 1. Archives of Ophthalmology (Chicago, Ill. : 1960). 128: 1129-35. PMID 20837795 DOI: 10.1001/archophthalmol.2010.201  1
2010 Koilkonda RD, Chou TH, Porciatti V, Hauswirth WW, Guy J. 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. Archives of Ophthalmology (Chicago, Ill. : 1960). 128: 876-83. PMID 20625049 DOI: 10.1001/archophthalmol.2010.135  1
2010 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. Molecular anthropology meets genetic medicine to treat blindness in the North African Jewish population: human gene therapy initiated in Israel. Human Gene Therapy. 21: 1749-57. PMID 20604683 DOI: 10.1089/hum.2010.047  1
2010 Boye SE, Boye SL, Pang J, Ryals R, Everhart D, Umino Y, Neeley AW, Besharse J, Barlow R, Hauswirth WW. Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse. Plos One. 5: e11306. PMID 20593011 DOI: 10.1371/journal.pone.0011306  1
2010 Lobanova ES, Herrmann R, Finkelstein S, Reidel B, Skiba NP, Deng WT, Jo R, Weiss ER, Hauswirth WW, Arshavsky VY. Mechanistic basis for the failure of cone transducin to translocate: why cones are never blinded by light. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 30: 6815-24. PMID 20484624 DOI: 10.1523/JNEUROSCI.0613-10.2010  1
2010 Caruso RC, Aleman TS, Cideciyan AV, Roman AJ, Sumaroka A, Mullins CL, Boye SL, Hauswirth WW, Jacobson SG. Retinal disease in Rpe65-deficient mice: comparison to human leber congenital amaurosis due to RPE65 mutations. Investigative Ophthalmology & Visual Science. 51: 5304-13. PMID 20484585 DOI: 10.1167/iovs.10-5559  1
2010 Li Q, Verma A, Han PY, Nakagawa T, Johnson RJ, Grant MB, Campbell-Thompson M, Jarajapu YP, Lei B, Hauswirth WW. Diabetic eNOS-knockout mice develop accelerated retinopathy. Investigative Ophthalmology & Visual Science. 51: 5240-6. PMID 20435587 DOI: 10.1167/iovs.09-5147  1
2010 Beltran WA, Boye SL, Boye SE, Chiodo VA, Lewin AS, Hauswirth WW, Aguirre GD. rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters. Gene Therapy. 17: 1162-74. PMID 20428215 DOI: 10.1038/gt.2010.56  1
2010 Komáromy AM, Alexander JJ, Rowlan JS, Garcia MM, Chiodo VA, Kaya A, Tanaka JC, Acland GM, Hauswirth WW, Aguirre GD. Gene therapy rescues cone function in congenital achromatopsia. Human Molecular Genetics. 19: 2581-93. PMID 20378608 DOI: 10.1093/hmg/ddq136  1
2010 Pang JJ, Alexander J, Lei B, Deng W, Zhang K, Li Q, Chang B, Hauswirth WW. Achromatopsia as a potential candidate for gene therapy. Advances in Experimental Medicine and Biology. 664: 639-46. PMID 20238068 DOI: 10.1007/978-1-4419-1399-9_73  1
2010 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. Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency. Gene Therapy. 17: 815-26. PMID 20237510 DOI: 10.1038/gt.2010.29  1
2010 Gorbatyuk MS, Knox T, LaVail MM, Gorbatyuk OS, Noorwez SM, Hauswirth WW, Lin JH, Muzyczka N, Lewin AS. Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78. Proceedings of the National Academy of Sciences of the United States of America. 107: 5961-6. PMID 20231467 DOI: 10.1073/pnas.0911991107  1
2010 Kong F, Li W, Li X, Zheng Q, Dai X, Zhou X, Boye SL, Hauswirth WW, Qu J, Pang JJ. Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse. Experimental Eye Research. 90: 546-54. PMID 20138034 DOI: 10.1016/j.exer.2010.01.011  1
2010 Qi Y, Liu X, Li H, Shenoy V, Li Q, Hauswirth WW, Sumners C, Katovich MJ. Selective tropism of the recombinant adeno-associated virus 9 serotype for rat cardiac tissue. The Journal of Gene Medicine. 12: 22-34. PMID 19830780 DOI: 10.1002/jgm.1404  1
2009 Koilkonda RD, Hauswirth WW, Guy J. Efficient expression of self-complementary AAV in ganglion cells of the ex vivo primate retina. Molecular Vision. 15: 2796-802. PMID 20019878  1
2009 Deng WT, Sakurai K, Liu J, Dinculescu A, Li J, Pang J, Min SH, Chiodo VA, Boye SL, Chang B, Kefalov VJ, Hauswirth WW. Functional interchangeability of rod and cone transducin alpha-subunits. Proceedings of the National Academy of Sciences of the United States of America. 106: 17681-6. PMID 19815523 DOI: 10.1073/pnas.0901382106  1
2009 Mancuso K, Hauswirth WW, Li Q, Connor TB, Kuchenbecker JA, Mauck MC, Neitz J, Neitz M. Gene therapy for red-green colour blindness in adult primates. Nature. 461: 784-7. PMID 19759534 DOI: 10.1038/nature08401  1
2009 Petersen-Jones SM, Bartoe JT, Fischer AJ, Scott M, Boye SL, Chiodo V, Hauswirth WW. AAV retinal transduction in a large animal model species: comparison of a self-complementary AAV2/5 with a single-stranded AAV2/5 vector. Molecular Vision. 15: 1835-42. PMID 19756181  1
2009 Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, Windsor EA, Conlon TJ, Sumaroka A, Roman AJ, Byrne BJ, Jacobson SG. Vision 1 year after gene therapy for Leber's congenital amaurosis. The New England Journal of Medicine. 361: 725-7. PMID 19675341 DOI: 10.1056/NEJMc0903652  1
2009 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. Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Human Gene Therapy. 20: 999-1004. PMID 19583479 DOI: 10.1089/hum.2009.086  1
2009 Tian G, Zhou Y, Hajkova D, Miyagi M, Dinculescu A, Hauswirth WW, Palczewski K, Geng R, Alagramam KN, Isosomppi J, Sankila EM, Flannery JG, Imanishi Y. Clarin-1, encoded by the Usher Syndrome III causative gene, forms a membranous microdomain: possible role of clarin-1 in organizing the actin cytoskeleton. The Journal of Biological Chemistry. 284: 18980-93. PMID 19423712 DOI: 10.1074/jbc.M109.003160  1
2009 Guy J, Qi X, Koilkonda RD, Arguello T, Chou TH, Ruggeri M, Porciatti V, Lewin AS, Hauswirth WW. Efficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual system. Investigative Ophthalmology & Visual Science. 50: 4205-14. PMID 19387075 DOI: 10.1167/iovs.08-3214  1
2009 Kosugi T, Heinig M, Nakayama T, Connor T, Yuzawa Y, Li Q, Hauswirth WW, Grant MB, Croker BP, Campbell-Thompson M, Zhang L, Atkinson MA, Segal MS, Nakagawa T. Lowering blood pressure blocks mesangiolysis and mesangial nodules, but not tubulointerstitial injury, in diabetic eNOS knockout mice. The American Journal of Pathology. 174: 1221-9. PMID 19246639 DOI: 10.2353/ajpath.2009.080605  1
2009 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. Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye. Molecular Vision. 15: 267-75. PMID 19190735  1
2009 Petrs-Silva H, Dinculescu A, Li Q, Min SH, Chiodo V, Pang JJ, Zhong L, Zolotukhin S, Srivastava A, Lewin AS, Hauswirth WW. High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors. Molecular Therapy : the Journal of the American Society of Gene Therapy. 17: 463-71. PMID 19066593 DOI: 10.1038/mt.2008.269  1
2009 Pease ME, Zack DJ, Berlinicke C, Bloom K, Cone F, Wang Y, Klein RL, Hauswirth WW, Quigley HA. Effect of CNTF on retinal ganglion cell survival in experimental glaucoma. Investigative Ophthalmology & Visual Science. 50: 2194-200. PMID 19060281 DOI: 10.1167/iovs.08-3013  1
2009 Zadro-Lamoureux LA, Zacks DN, Baker AN, Zheng QD, Hauswirth WW, Tsilfidis C. XIAP effects on retinal detachment-induced photoreceptor apoptosis [corrected]. Investigative Ophthalmology & Visual Science. 50: 1448-53. PMID 19060276 DOI: 10.1167/iovs.08-2855  1
2009 Mu W, Long DA, Ouyang X, Agarwal A, Cruz PE, Roncal CA, Nakagawa T, Yu X, Hauswirth WW, Johnson RJ. Angiostatin overexpression is associated with an improvement in chronic kidney injury by an anti-inflammatory mechanism. American Journal of Physiology. Renal Physiology. 296: F145-52. PMID 18971211 DOI: 10.1152/ajprenal.90430.2008  1
2009 Pechan P, Rubin H, Lukason M, Ardinger J, DuFresne E, Hauswirth WW, Wadsworth SC, Scaria A. Novel anti-VEGF chimeric molecules delivered by AAV vectors for inhibition of retinal neovascularization. Gene Therapy. 16: 10-6. PMID 18633446 DOI: 10.1038/gt.2008.115  1
2008 Liu J, Saghizadeh M, Tuli SS, Kramerov AA, Lewin AS, Bloom DC, Hauswirth WW, Castro MG, Schultz GS, Ljubimov AV. Different tropism of adenoviruses and adeno-associated viruses to corneal cells: implications for corneal gene therapy. Molecular Vision. 14: 2087-96. PMID 19023450  1
2008 Li Q, Miller R, Han PY, Pang J, Dinculescu A, Chiodo V, Hauswirth WW. Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential Molecular Vision. 14: 1760-1769. PMID 18836574  1
2008 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, ... ... Hauswirth WW, et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proceedings of the National Academy of Sciences of the United States of America. 105: 15112-7. PMID 18809924 DOI: 10.1073/pnas.0807027105  1
2008 Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG. 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. Human Gene Therapy. 19: 979-90. PMID 18774912 DOI: 10.1089/hum.2008.107  1
2008 Li Q, Dinculescu A, Shan Z, Miller R, Pang J, Lewin AS, Raizada MK, Hauswirth WW. Downregulation of p22phox in retinal pigment epithelial cells inhibits choroidal neovascularization in mice Molecular Therapy. 16: 1688-1694. PMID 18665154 DOI: 10.1038/mt.2008.164  1
2008 Mauck MC, Mancuso K, Kuchenbecker JA, Connor TB, Hauswirth WW, Neitz J, Neitz M. Longitudinal evaluation of expression of virally delivered transgenes in gerbil cone photoreceptors. Visual Neuroscience. 25: 273-82. PMID 18598398 DOI: 10.1017/S0952523808080577  1
2008 Alexander JJ, Hauswirth WW. Prospects for retinal cone-targeted gene therapy Drug News and Perspectives. 21: 267-271. PMID 18596991 DOI: 10.1358/dnp.2008.21.5.1219012  1
2008 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. AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation. Investigative Ophthalmology & Visual Science. 49: 4278-83. PMID 18586879 DOI: 10.1167/iovs.07-1622  1
2008 Damiani D, Alexander JJ, O'Rourke JR, McManus M, Jadhav AP, Cepko CL, Hauswirth WW, Harfe BD, Strettoi E. Dicer inactivation leads to progressive functional and structural degeneration of the mouse retina. The Journal of Neuroscience : the Official Journal of the Society For Neuroscience. 28: 4878-87. PMID 18463241 DOI: 10.1523/JNEUROSCI.0828-08.2008  1
2008 Janssen A, Min SH, Molday LL, Tanimoto N, Seeliger MW, Hauswirth WW, Molday RS, Weber BHF. Effect of Late-stage therapy on disease progression in AAV-mediated rescue of photoreceptor cells in the retinoschisin-deficient mouse Molecular Therapy. 16: 1010-1017. PMID 18388913 DOI: 10.1038/mt.2008.57  1
2008 Komáromy AM, Alexander JJ, Cooper AE, Chiodo VA, Glushakova LG, Acland GM, Hauswirth WW, Aguirre GD. Targeting gene expression to cones with human cone opsin promoters in recombinant AAV. Gene Therapy. 15: 1049-55. PMID 18337838 DOI: 10.1038/gt.2008.32  1
2008 Raisler BJ, Nozaki M, Baffi J, Hauswirth WW, Ambati J. Toward a higher fidelity model of AMD. Advances in Experimental Medicine and Biology. 613: 185-92. PMID 18188944 DOI: 10.1007/978-0-387-74904-4_21  1
2008 Pang Jj, Lauramore A, Deng Wt, Li Q, Doyle TJ, Chiodo V, Li J, Hauswirth WW. Comparative analysis of in vivo and in vitro AAV vector transduction in the neonatal mouse retina: Effects of serotype and site of administration Vision Research. 48: 377-385. PMID 17950399 DOI: 10.1016/j.visres.2007.08.009  1
2008 Li Q, Timmers AM, Guy J, Pang J, Hauswirth WW. Cone-specific expression using a human red opsin promoter in recombinant AAV Vision Research. 48: 332-338. PMID 17905404 DOI: 10.1016/j.visres.2007.07.026  1
2008 Alexander JJ, Hauswirth WW. Adeno-associated viral vectors and the retina Advances in Experimental Medicine and Biology. 613: 121-128. DOI: 10.1007/978-0-387-74904-4_13  1
2008 Gorbatyuk MS, Hauswirth WW, Lewin AS. Gene therapy for mouse models of adrp Advances in Experimental Medicine and Biology. 613: 107-112. DOI: 10.1007/978-0-387-74904-4_11  1
2007 Andino LM, Conlon TJ, Porvasnik SL, Boye SL, Hauswirth WW, Lewin AS. Rapid, widespread transduction of the murine myocardium using self-complementary Adeno-associated virus. Genetic Vaccines and Therapy. 5: 13. PMID 18070352 DOI: 10.1186/1479-0556-5-13  1
2007 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. Intraocular CNTF reduces vision in normal rats in a dose-dependent manner. Investigative Ophthalmology & Visual Science. 48: 5756-66. PMID 18055829 DOI: 10.1167/iovs.07-0054  1
2007 Qi X, Sun L, Lewin AS, Hauswirth WW, Guy J. Long-term suppression of neurodegeneration in chronic experimental optic neuritis: Antioxidant gene therapy Investigative Ophthalmology and Visual Science. 48: 5360-5370. PMID 18055782 DOI: 10.1167/iovs.07-0254  1
2007 Roman AJ, Boye SL, Aleman TS, Pang JJ, McDowell JH, Boye SE, Cideciyan AV, Jacobson SG, Hauswirth WW. Electroretinographic analyses of Rpe65-mutant rd12 mice: developing an in vivo bioassay for human gene therapy trials of Leber congenital amaurosis. Molecular Vision. 13: 1701-10. PMID 17960108  1
2007 Justilien V, Pang JJ, Renganathan K, Zhan X, Crabb JW, Kim SR, Sparrow JR, Hauswirth WW, Lewin AS. SOD2 knockdown mouse model of early AMD. Investigative Ophthalmology & Visual Science. 48: 4407-20. PMID 17898259 DOI: 10.1167/iovs.07-0432  1
2007 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. Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation. Plos Medicine. 4: e230. PMID 17594175 DOI: 10.1371/journal.pmed.0040230  1
2007 Alexander JJ, Umino Y, Everhart D, Chang B, Min SH, Li Q, Timmers AM, Hawes NL, Pang JJ, Barlow RB, Hauswirth WW. Restoration of cone vision in a mouse model of achromatopsia. Nature Medicine. 13: 685-7. PMID 17515894 DOI: 10.1038/nm1596  1
2007 Kwon BK, Liu J, Lam C, Plunet W, Oschipok LW, Hauswirth W, Di Polo A, Blesch A, Tetzlaff W. 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. Spine. 32: 1164-73. PMID 17495772 DOI: 10.1097/BRS.0b013e318053ec35  1
2007 Aarnisalo AA, Pietola L, Joensuu J, Isosomppi J, Aarnisalo P, Dinculescu A, Lewin AS, Flannery J, Hauswirth WW, Sankila EM, Jero J. Anti-clarin-1 AAV-delivered ribozyme induced apoptosis in the mouse cochlea Hearing Research. 230: 9-16. PMID 17493778 DOI: 10.1016/j.heares.2007.03.004  1
2007 White DA, Fritz JJ, Hauswirth WW, Kaushal S, Lewin AS. Increased sensitivity to light-induced damage in a mouse model of autosomal dominant retinal disease. Investigative Ophthalmology & Visual Science. 48: 1942-51. PMID 17460245 DOI: 10.1167/iovs.06-1131  1
2007 Leonard KC, Petrin D, Coupland SG, Baker AN, Leonard BC, LaCasse EC, Hauswirth WW, Komeluk RG, Tsilfidis C. XIAP protection of photoreceptors in animal models of retinitis pigmentosa Plos One. 2. PMID 17375200 DOI: 10.1371/journal.pone.0000314  1
2007 Gorbatyuk M, Justilien V, Liu J, Hauswirth WW, Lewin AS. Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery Vision Research. 47: 1202-1208. PMID 17292939 DOI: 10.1016/j.visres.2006.11.026  1
2006 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, ... ... Hauswirth WW, et al. Proceedings of the Third International Symposium on Retinopathy of Prematurity: an update on ROP from the lab to the nursery (November 2003, Anaheim, California). Molecular Vision. 12: 532-80. PMID 16735995  1
2005 Deng WT, Yan Z, Dinculescu A, Pang J, Teusner JT, Cortez NG, Berns KI, Hauswirth WW. Adeno-associated virus-mediated expression of vascular endothelial growth factor peptides inhibits retinal neovascularization in a mouse model of oxygen-induced retinopathy. Human Gene Therapy. 16: 1247-54. PMID 16259558 DOI: 10.1089/hum.2005.16.1247  1
2005 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, et al. Interleukin 10 attenuates neointimal proliferation and inflammation in aortic allografts by a heme oxygenase-dependent pathway. Proceedings of the National Academy of Sciences of the United States of America. 102: 7251-6. PMID 15878989 DOI: 10.1073/pnas.0502407102  1
2005 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. Efficient transduction of vascular endothelial cells with recombinant adeno-associated virus serotype 1 and 5 vectors. Human Gene Therapy. 16: 235-47. PMID 15761263 DOI: 10.1089/hum.2005.16.235  1
2004 Fritz JJ, Gorbatyuk M, Lewin AS, Hauswirth WW. Design and validation of therapeutic hammerhead ribozymes for autosomal dominant diseases. Methods in Molecular Biology (Clifton, N.J.). 252: 221-36. PMID 15017052 DOI: 10.1385/1-59259-746-7:221  1
2004 Hauswirth WW, Li Q, Raisler B, Timmers AM, Berns KI, Flannery JG, LaVail MM, Lewin AS. Range of retinal diseases potentially treatable by AAV-vectored gene therapy. Novartis Foundation Symposium. 255: 179-88; discussion 1. PMID 14750604  1
2003 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. Gene delivery in renal tubular epithelial cells using recombinant adeno-associated viral vectors. Journal of the American Society of Nephrology : Jasn. 14: 947-58. PMID 12660329 DOI: 10.1097/01.ASN.0000057858.45649.F7  1
2002 Raisler BJ, Berns KI, Grant MB, Beliaev D, Hauswirth WW. Adeno-associated virus type-2 expression of pigmented epithelium-derived factor or Kringles 1-3 of angiostatin reduce retinal neovascularization. Proceedings of the National Academy of Sciences of the United States of America. 99: 8909-14. PMID 12072560 DOI: 10.1073/pnas.122247299  1
2002 Mori K, Gehlbach P, Yamamoto S, Duh E, Zack DJ, Li Q, Berns KI, Raisler BJ, Hauswirth WW, Campochiaro PA. AAV-mediated gene transfer of pigment epithelium-derived factor inhibits choroidal neovascularization. Investigative Ophthalmology & Visual Science. 43: 1994-2000. PMID 12037010  1
2002 Fritz JJ, White DA, Lewin AS, Hauswirth WW. Designing and characterizing hammerhead ribozymes for use in AAV vector-mediated retinal gene therapies. Methods in Enzymology. 346: 358-77. PMID 11883079  0.64
2001 Hauswirth WW, Shaw LC, Whalen PO, Fritz JJ, White DA, Lewin AS. Inhibition of gene expression by ribozymes. Methods in Molecular Medicine. 47: 105-24. PMID 21394581 DOI: 10.1385/1-59259-085-3:105  0.64
1980 Cheung AK, Hoggan MD, Hauswirth WW, Berns KI. Integration of the adeno-associated virus genome into cellular DNA in latently infected human Detroit 6 cells. Journal of Virology. 33: 739-48. PMID 6251245  1
1979 Berns KI, Hauswirth WW. Adeno-associated viruses. Advances in Virus Research. 25: 407-49. PMID 230727  1
1979 Berns KI, Hauswirth WW, Fife KH, Lusby E. Adeno-associated virus DNA replication. Cold Spring Harbor Symposia On Quantitative Biology. 43: 781-7. PMID 226321  1
1979 Hauswirth WW, Berns KI. Adeno-associated virus DNA replication: nonunit-length molecules. Virology. 93: 57-68. PMID 219605 DOI: 10.1016/0042-6822(79)90275-7  1
1977 Spear IS, Fife KH, Hauswirth WW, Jones CJ, Berns KI. Evidence for two nucleotide sequence orientations within the terminal repetition of adeno-associated virus DNA. Journal of Virology. 24: 627-34. PMID 916029  1
1977 Hauswirth WW, Berns KI. Origin and termination of adeno-associated virus DNA replication. Virology. 78: 488-99. PMID 867815 DOI: 10.1016/0042-6822(77)90125-8  1
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