| Year |
Citation |
Score |
| 2023 |
Araujo VG, Dias MS, Hauswirth WW, Linden R, Petrs-Silva H. rAAV-compatible human mini promoters enhance transgene expression in rat retinal ganglion cells. Experimental Eye Research. 239: 109758. PMID 38123011 DOI: 10.1016/j.exer.2023.109758 |
0.649 |
|
| 2023 |
Yan B, Viswanathan S, Brodie SE, Deng WT, Coleman KE, Hauswirth WW, Nirenberg S. A clinically viable approach to restoring visual function using optogenetic gene therapy. Molecular Therapy. Methods & Clinical Development. 29: 406-417. PMID 37251979 DOI: 10.1016/j.omtm.2023.05.005 |
0.366 |
|
| 2023 |
Occelli LM, Zobel L, Stoddard J, Wagner J, Pasmanter N, Querubin J, Renner LM, Reynaga R, Winkler PA, Sun K, Felipe Lp Marinho L, O'Riordan CR, Frederick A, Lauer A, Tsang SH, ... Hauswirth WW, et al. Development of a translatable gene augmentation therapy for CNGB1-Retinitis Pigmentosa. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 37056049 DOI: 10.1016/j.ymthe.2023.04.005 |
0.426 |
|
| 2022 |
Liu Y, Eastwood JD, Alba DE, Velmurugan S, Sun N, Porciatti V, Lee RK, Hauswirth WW, Guy J, Yu H. Gene therapy restores mitochondrial function and protects retinal ganglion cells in optic neuropathy induced by a mito-targeted mutant ND1 gene. Gene Therapy. PMID 35383288 DOI: 10.1038/s41434-022-00333-6 |
0.37 |
|
| 2022 |
Lani-Louzada R, Marra C, Dias MS, de Araújo VG, Abreu CA, Ribas VT, Adesse D, Allodi S, Chiodo V, Hauswirth W, Petrs-Silva H, Linden R. Neuroprotective Gene Therapy by Overexpression of the Transcription Factor MAX in Rat Models of Glaucomatous Neurodegeneration. Investigative Ophthalmology & Visual Science. 63: 5. PMID 35103748 DOI: 10.1167/iovs.63.2.5 |
0.547 |
|
| 2021 |
Aguirre GD, Cideciyan AV, Dufour VL, Ripolles-García A, Sudharsan R, Swider M, Nikonov R, Iwabe S, Boye SL, Hauswirth WW, Jacobson SG, Beltran WA. Gene therapy reforms photoreceptor structure and restores vision in NPHP5-associated Leber congenital amaurosis. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 34715016 DOI: 10.1016/j.ymthe.2021.10.021 |
0.306 |
|
| 2021 |
Zhu P, Dyka F, Ma X, Yin L, Yu H, Baehr W, Hauswirth WW, Deng WT. Disease mechanisms of X-linked cone dystrophy caused by missense mutations in the red and green cone opsins. Faseb Journal : Official Publication of the Federation of American Societies For Experimental Biology. 35: e21927. PMID 34547123 DOI: 10.1096/fj.202101066R |
0.324 |
|
| 2021 |
Visuvanathan S, Baker AN, Lagali PS, Coupland SG, Miller G, Hauswirth WW, Tsilfidis C. XIAP gene therapy effects on retinal ganglion cell structure and function in a mouse model of glaucoma. Gene Therapy. PMID 34363035 DOI: 10.1038/s41434-021-00281-7 |
0.335 |
|
| 2021 |
Aguirre GD, Cideciyan AV, Dufour VL, Ripolles-García A, Sudharsan R, Swider M, Nikonov R, Iwabe S, Boye SL, Hauswirth WW, Jacobson SG, Beltran WA. Gene Therapy Reforms Photoreceptor Structure and Restores Vision in NPHP5-associated Leber Congenital Amaurosis. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 33781914 DOI: 10.1016/j.ymthe.2021.03.021 |
0.403 |
|
| 2020 |
Wassmer SJ, De Repentigny Y, Sheppard D, Lagali PS, Fang L, Coupland SG, Kothary R, Guy J, Hauswirth WW, Tsilfidis C. XIAP Protects Retinal Ganglion Cells in the Mutant ND4 Mouse Model of Leber Hereditary Optic Neuropathy. Investigative Ophthalmology & Visual Science. 61: 49. PMID 32735323 DOI: 10.1167/Iovs.61.8.49 |
0.454 |
|
| 2020 |
Goodson NB, Park KU, Silver JS, Chiodo VA, Hauswirth WW, Brzezinski JA. Prdm1 overexpression causes a photoreceptor fate-shift in nascent, but not mature, bipolar cells. Developmental Biology. PMID 32562755 DOI: 10.1016/J.Ydbio.2020.06.003 |
0.358 |
|
| 2020 |
Ross M, Obolensky A, Averbukh E, Ezra-Elia R, Yamin E, Honig H, Dvir H, Rosov A, Hauswirth WW, Gootwine E, Banin E, Ofri R. Evaluation of photoreceptor transduction efficacy of capsid-modified AAV vectors following intravitreal and subretinal delivery in sheep. Human Gene Therapy. PMID 32486858 DOI: 10.1089/Hum.2020.023 |
0.51 |
|
| 2020 |
Litts KM, Georgiou M, Langlo CS, Patterson EJ, Mastey RR, Kalitzeos A, Linderman RE, Lam BL, Fishman GA, Pennesi ME, Kay CN, Hauswirth WW, Michaelides M, Carroll J. Interocular symmetry of foveal cone topography in congenital achromatopsia. Current Eye Research. PMID 32108519 DOI: 10.1080/02713683.2020.1737138 |
0.306 |
|
| 2019 |
Wang H, Kunz E, Stoddard GJ, Hauswirth WW, Hartnett ME. Optimal Inhibition of Choroidal Neovascularization by scAAV2 with VMD2 Promoter-driven Active Rap1a in the RPE. Scientific Reports. 9: 15732. PMID 31673119 DOI: 10.1038/S41598-019-52163-Z |
0.452 |
|
| 2019 |
Xu L, Bolch SN, Santiago CP, Dyka FM, Akil O, Lobanova ES, Wang Y, Martemyanov KA, Hauswirth WW, Smith WC, Handa JT, Blackshaw S, Ash JD, Dinculescu A. Clarin-1 expression in adult mouse and human retina highlights a role of Müller glia in Usher syndrome. The Journal of Pathology. PMID 31625146 DOI: 10.1002/Path.5360 |
0.464 |
|
| 2019 |
Gardiner KL, Cideciyan AV, Swider M, Dufour VL, Sumaroka A, Komáromy AM, Hauswirth WW, Iwabe S, Jacobson SG, Beltran WA, Aguirre GD. Long-Term Structural Outcomes of Late-Stage RPE65 Gene Therapy. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 31604676 DOI: 10.1016/J.Ymthe.2019.08.013 |
0.445 |
|
| 2019 |
Dias MS, Araujo VG, Vasconcelos T, Li Q, Hauswirth WW, Linden R, Petrs-Silva H. Retina transduction by rAAV2 after intravitreal injection: comparison between mouse and rat. Gene Therapy. PMID 31562387 DOI: 10.1038/S41434-019-0100-9 |
0.643 |
|
| 2019 |
Deng WT, Li J, Zhu P, Freedman B, Smith WC, Baehr W, Hauswirth WW. Rescue of M-cone Function in Aged Opn1mw-/- Mice, a Model for Late-Stage Blue Cone Monochromacy. Investigative Ophthalmology & Visual Science. 60: 3644-3651. PMID 31469404 DOI: 10.1167/Iovs.19-27079 |
0.346 |
|
| 2019 |
Dyka FM, Molday LL, Chiodo V, Molday RS, Hauswirth WW. Dual ABCA4-AAV vector treatment reduces pathogenic retinal A2E accumulation in a mouse model of autosomal recessive Stargardt Disease. Human Gene Therapy. PMID 31418294 DOI: 10.1089/Hum.2019.132 |
0.506 |
|
| 2019 |
Nascimento-Dos-Santos G, Teixeira-Pinheiro LC, da Silva-Júnior AJ, Carvalho LRP, Mesentier-Louro LA, Hauswirth WW, Mendez-Otero R, Santiago MF, Petrs-Silva H. Effects of a combinatorial treatment with gene and cell therapy on retinal ganglion cell survival and axonal outgrowth after optic nerve injury. Gene Therapy. PMID 31243393 DOI: 10.1038/S41434-019-0089-0 |
0.382 |
|
| 2019 |
Akil O, Dyka F, Calvet C, Emptoz A, Lahlou G, Nouaille S, Boutet de Monvel J, Hardelin JP, Hauswirth WW, Avan P, Petit C, Safieddine S, Lustig LR. Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model. Proceedings of the National Academy of Sciences of the United States of America. PMID 30782832 DOI: 10.1073/Pnas.1817537116 |
0.475 |
|
| 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 |
0.332 |
|
| 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 |
0.504 |
|
| 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 |
0.487 |
|
| 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 |
0.557 |
|
| 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 |
0.442 |
|
| 2018 |
Ofri R, Averbukh E, Ezra-Elia R, Ross M, Honig H, Obolensky A, Rosov A, Hauswirth WW, Gootwine E, Banin E. Six Years and Counting: Restoration of Photopic Retinal Function and Visual Behavior Following Gene Augmentation Therapy in a Sheep Model of CNGA3 Achromatopsia. Human Gene Therapy. PMID 29926749 DOI: 10.1089/Hum.2018.076 |
0.488 |
|
| 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 |
0.402 |
|
| 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 |
0.416 |
|
| 2018 |
Yu H, Porciatti V, Lewin A, Hauswirth W, Guy J. Longterm Reversal of Severe Visual Loss by Mitochondrial Gene Transfer in a Mouse Model of Leber Hereditary Optic Neuropathy. Scientific Reports. 8: 5587. PMID 29615737 DOI: 10.1038/S41598-018-23836-Y |
0.42 |
|
| 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 |
0.346 |
|
| 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 |
0.514 |
|
| 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 |
0.39 |
|
| 2018 |
Bosco A, Anderson S, Breen K, Romero C, Steele M, Chiodo V, Boyle S, Hauswirth W, Tomlinson S, Vetter M. C3-targeted retinal gene therapy limits neurodegeneration onset and progression in age-related mouse glaucoma Molecular Immunology. 102: 136. DOI: 10.1016/J.Molimm.2018.06.035 |
0.463 |
|
| 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 |
0.494 |
|
| 2017 |
Geng R, Omar A, Gopal SR, Chen DH, Stepanyan R, Basch ML, Dinculescu A, Furness DN, Saperstein D, Hauswirth W, Lustig LR, Alagramam KN. Modeling and Preventing Progressive Hearing Loss in Usher Syndrome III. Scientific Reports. 7: 13480. PMID 29044151 DOI: 10.1038/S41598-017-13620-9 |
0.376 |
|
| 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 |
0.448 |
|
| 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 |
0.506 |
|
| 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 |
0.692 |
|
| 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 |
0.431 |
|
| 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 |
0.562 |
|
| 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 |
0.33 |
|
| 2017 |
Reid CA, Boye SL, Hauswirth WW, Lipinski DM. miRNA-mediated post-transcriptional silencing of transgenes leads to increased adeno-associated viral vector yield and targeting specificity. Gene Therapy. 24: 462-469. PMID 28617420 DOI: 10.1038/Gt.2017.50 |
0.335 |
|
| 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 |
0.746 |
|
| 2017 |
Wassmer S, Leonard B, Coupland S, Baker A, Hamilton J, Hauswirth WW, Tsilfidis C. Over-expression of the X-linked Inhibitor of Apoptosis Protects Against Retinal Degeneration in a Feline Model of Retinal Detachment. Human Gene Therapy. PMID 28335619 DOI: 10.1089/Hum.2016.161 |
0.489 |
|
| 2017 |
Gootwine E, Abu-Siam M, Obolensky A, Rosov A, Honig H, Nitzan T, Shirak A, Ezra-Elia R, Yamin E, Banin E, Averbukh E, Hauswirth WW, Ofri R, Seroussi E. Gene Augmentation Therapy for a Missense Substitution in the cGMP-Binding Domain of Ovine CNGA3 Gene Restores Vision in Day-Blind Sheep. Investigative Ophthalmology & Visual Science. 58: 1577-1584. PMID 28282490 DOI: 10.1167/Iovs.16-20986 |
0.388 |
|
| 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 |
0.555 |
|
| 2017 |
Langlo CS, Erker LR, Parker M, Patterson EJ, Higgins BP, Summerfelt P, Razeen MM, Collison FT, Fishman GA, Kay CN, Zhang J, Weleber RG, Yang P, Pennesi ME, Lam BL, ... ... Hauswirth WW, et al. REPEATABILITY AND LONGITUDINAL ASSESSMENT OF FOVEAL CONE STRUCTURE IN CNGB3-ASSOCIATED ACHROMATOPSIA. Retina (Philadelphia, Pa.). PMID 28145975 DOI: 10.1097/Iae.0000000000001434 |
0.312 |
|
| 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 |
0.433 |
|
| 2017 |
Vetter M, Bosco A, Anderson S, Breen K, Romero C, Steele M, Chiodo V, Boye S, Hauswirth W, Tomlinson S. Contribution of microglia and complement activation to glaucoma progression Acta Ophthalmologica. 95. DOI: 10.1111/J.1755-3768.2017.03621 |
0.62 |
|
| 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 |
0.438 |
|
| 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 |
0.521 |
|
| 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 |
0.437 |
|
| 2016 |
Langouet-Astrie CJ, Yang Z, Polisetti SM, Welsbie DS, Hauswirth WW, Zack DJ, Merbs SL, Enke RA. Characterization of intravitreally delivered capsid mutant AAV2-Cre vector to induce tissue-specific mutations in murine retinal ganglion cells. Experimental Eye Research. PMID 27481653 DOI: 10.1016/J.Exer.2016.07.019 |
0.529 |
|
| 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 |
0.419 |
|
| 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 |
0.727 |
|
| 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 |
0.349 |
|
| 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 |
0.501 |
|
| 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 |
0.694 |
|
| 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 |
0.764 |
|
| 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 |
0.466 |
|
| 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 |
0.352 |
|
| 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 |
0.344 |
|
| 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 |
0.552 |
|
| 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 |
0.662 |
|
| 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 |
0.466 |
|
| 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 |
0.535 |
|
| 2016 |
Ye G, Budzynski E, Sonnentag P, Nork TM, Miller PE, McPherson L, Hoeve JNV, Smith LM, Arndt TA, Mandapati S, Robinson PM, Calcedo R, Knop DR, Hauswirth WW, Chulay JD. 299. Safety and Biodistribution Study of rAAV2tYF-PR1.7-hCNGB3 in CNGB3-Deficient Mice Molecular Therapy. 24: S120. DOI: 10.1016/S1525-0016(16)33108-2 |
0.43 |
|
| 2016 |
Ye G, Budzynski E, Sonnentag P, Nork TM, Miller PE, Sharma AK, Hoeve JNV, Smith LM, Arndt T, Calcedo R, Gaskin C, Robinson PM, Knop DR, Hauswirth WW, Chulay JD. 88. Safety and Biodistribution Study of rAAV2tYF-PR1.7-hCNGB3 in Nonhuman Primates Molecular Therapy. 24: S38-S39. DOI: 10.1016/S1525-0016(16)32897-0 |
0.421 |
|
| 2015 |
Jara JH, Stanford MJ, Zhu Y, Tu M, Hauswirth WW, Bohn MC, DeVries SH, Özdinler PH. Healthy and diseased corticospinal motor neurons are selectively transduced upon direct AAV2-2 injection into the motor cortex. Gene Therapy. PMID 26704722 DOI: 10.1038/Gt.2015.112 |
0.316 |
|
| 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 |
0.552 |
|
| 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 |
0.733 |
|
| 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 |
0.479 |
|
| 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 |
0.754 |
|
| 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 |
0.531 |
|
| 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 |
0.435 |
|
| 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 |
0.689 |
|
| 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 |
0.747 |
|
| 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 |
0.503 |
|
| 2015 |
Jiang L, Tam BM, Ying G, Wu S, Hauswirth WW, Frederick JM, Moritz OL, Baehr W. Kinesin family 17 (osmotic avoidance abnormal-3) is dispensable for photoreceptor morphology and function. Faseb Journal : Official Publication of the Federation of American Societies For Experimental Biology. PMID 26229057 DOI: 10.1096/Fj.15-275677 |
0.396 |
|
| 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 |
0.553 |
|
| 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 |
0.382 |
|
| 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 |
0.725 |
|
| 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 |
0.517 |
|
| 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 |
0.433 |
|
| 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 |
0.528 |
|
| 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 |
0.708 |
|
| 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 |
0.458 |
|
| 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 |
0.328 |
|
| 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 |
0.676 |
|
| 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 |
0.47 |
|
| 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 |
0.415 |
|
| 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 |
0.697 |
|
| 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 |
0.502 |
|
| 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 |
0.437 |
|
| 2015 |
Koilkonda RD, Hauswirth WW, Chiodo V, Boye SL, Gonzalez P, Guy J. 608. Evaluation of Pre-Existing Neutralizing Antibodies Against Tyrosine-Mutant AAV2 in Leber Hereditary Optic Neuropathy (LHON) Patients Molecular Therapy. 23: S241. DOI: 10.1016/S1525-0016(16)34217-4 |
0.364 |
|
| 2015 |
Hickmott JW, Chen C, Arenillas DJ, Li Y, Molday LL, Korecki AJ, Ling Lam S, Ling Lam S, Bonaguro RJ, Zhou M, Chan AY, Boye SL, Hauswirth WW, Molday RS, Wasserman WW, et al. 599. Deep Informatics Utilized to Design MiniPromoters for Driving PAX6-Like Retinal Expression with AAV Molecular Therapy. 23: S238. DOI: 10.1016/S1525-0016(16)34208-3 |
0.508 |
|
| 2015 |
Benskey MJ, Benskey MJ, Kuhn NC, Kuhn NC, Galligan JJ, Galligan JJ, Garcia J, Garcia J, Hauswirth WW, Hauswirth WW, Mueller C, Boye SL, Mnafredsson FP. 544. Targeted Gene Delivery To the Enteric Nervous System: α-Synuclein Impairs Colonic Motility Molecular Therapy. 23: S218. DOI: 10.1016/S1525-0016(16)34153-3 |
0.321 |
|
| 2015 |
Lipinski DM, Reid CA, Boye SL, Peterson JJ, Qi X, Boulton ME, Hauswirth WW. 314. Systemic Vascular Transduction Following Intravenous Injection of Capsid Mutant Adeno-Associated Virus Molecular Therapy. 23: S127. DOI: 10.1016/S1525-0016(16)33923-5 |
0.442 |
|
| 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 |
0.466 |
|
| 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 |
0.423 |
|
| 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 |
0.38 |
|
| 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 |
0.355 |
|
| 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 |
0.344 |
|
| 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 |
0.382 |
|
| 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 |
0.48 |
|
| 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 |
0.432 |
|
| 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 |
0.37 |
|
| 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 |
0.733 |
|
| 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 |
0.33 |
|
| 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 |
0.729 |
|
| 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 |
0.56 |
|
| 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 |
0.722 |
|
| 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 |
0.714 |
|
| 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 |
0.553 |
|
| 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 |
0.532 |
|
| 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 |
0.551 |
|
| 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 |
0.347 |
|
| 2014 |
Pang J, Deng W, 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. Correction: AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia Plos One. 9. DOI: 10.1371/Annotation/29Fb0Ebc-B1C7-4D05-Bd70-D29B10299Df4 |
0.327 |
|
| 2014 |
GUZIEWICZ K, BELTRAN W, CIDECIYAN A, KOMÁROMY A, IWABE S, DUTROW E, ZANGERL B, HAUSWIRTH W, JACOBSON S, AGUIRRE G. Gene therapy for bestrophinopathies Acta Ophthalmologica. 92: 0-0. DOI: 10.1111/J.1755-3768.2014.3264.X |
0.485 |
|
| 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 |
0.55 |
|
| 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 |
0.426 |
|
| 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 |
0.439 |
|
| 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 |
0.417 |
|
| 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 |
0.516 |
|
| 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 |
0.398 |
|
| 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 |
0.526 |
|
| 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 |
0.75 |
|
| 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 |
0.729 |
|
| 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 |
0.521 |
|
| 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 |
0.552 |
|
| 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 |
0.729 |
|
| 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 |
0.387 |
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| 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 |
0.368 |
|
| 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 |
0.714 |
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| 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 |
0.765 |
|
| 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 |
0.4 |
|
| 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 |
0.521 |
|
| 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 |
0.745 |
|
| 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. Correction: Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors Plos One. 8. DOI: 10.1371/Annotation/99Ee1789-A658-4Fb0-8593-40A40E9F344A |
0.676 |
|
| 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 |
0.364 |
|
| 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 |
0.76 |
|
| 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 |
0.34 |
|
| 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 |
0.309 |
|
| 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 |
0.519 |
|
| 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 |
0.395 |
|
| 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 |
0.467 |
|
| 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 |
0.311 |
|
| 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 |
0.705 |
|
| 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 |
0.495 |
|
| 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 |
0.548 |
|
| 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 |
0.557 |
|
| 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 |
0.507 |
|
| 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 |
0.379 |
|
| 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 |
0.545 |
|
| 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 |
0.522 |
|
| 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 |
0.396 |
|
| 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 |
0.55 |
|
| 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 |
0.492 |
|
| 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 |
0.346 |
|
| 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 |
0.427 |
|
| 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 |
0.475 |
|
| 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 |
0.301 |
|
| 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 |
0.754 |
|
| 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 |
0.444 |
|
| 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 |
0.567 |
|
| 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 |
0.739 |
|
| 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 |
0.681 |
|
| 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 |
0.74 |
|
| 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 |
0.478 |
|
| 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 |
0.525 |
|
| 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 |
0.462 |
|
| 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 |
0.312 |
|
| 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 |
0.546 |
|
| 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 |
0.523 |
|
| 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 |
0.756 |
|
| 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 |
0.496 |
|
| 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 |
0.488 |
|
| 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 |
0.461 |
|
| 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 |
0.364 |
|
| 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 |
0.408 |
|
| 2011 |
Komáromy AM, Alexander JJ, Cooper AE, Chiodo VA, Acland GM, Hauswirth WW, Aguirre GD. Erratum: Targeting gene expression to cones with human cone opsin promoters in recombinant AAV Gene Therapy. 18: 1179-1179. DOI: 10.1038/gt.2011.156 |
0.337 |
|
| 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 |
0.438 |
|
| 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 |
0.508 |
|
| 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 |
0.405 |
|
| 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 |
0.741 |
|
| 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 |
0.343 |
|
| 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 |
0.444 |
|
| 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 |
0.343 |
|
| 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 |
0.756 |
|
| 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 |
0.542 |
|
| 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 |
0.466 |
|
| 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 |
0.72 |
|
| 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 |
0.384 |
|
| 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 |
0.503 |
|
| 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 |
0.318 |
|
| 2010 |
Gingras G, Komaromy AM, Tseng B, Alexander JJ, Chiodo VV, Hauswirth WW, Acland GM, Aguirre GD, Brainard DH, Aguirre GK. Cortical recovery following gene therapy in a canine model of achromatopsia Journal of Vision. 9: 311-311. DOI: 10.1167/9.8.311 |
0.353 |
|
| 2010 |
Mancuso K, Hauswirth WW, Connor TB, Kuchenbecker JA, Mauck MC, Neitz J, Neitz M. Analysis of a primate cone mosaic following targeted gene transfer using recombinant adeno-associated virus vectors Journal of Vision. 8: 69-69. DOI: 10.1167/8.17.69 |
0.407 |
|
| 2010 |
Mancuso K, Neitz J, Hauswirth WW, Connor TB, Neitz M. Gene therapy treatment of color blindness in adult primates Journal of Vision. 7: 15-15. DOI: 10.1167/7.15.15 |
0.342 |
|
| 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 |
0.433 |
|
| 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 |
0.41 |
|
| 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 |
0.388 |
|
| 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 |
0.402 |
|
| 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 |
0.396 |
|
| 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 |
0.479 |
|
| 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 |
0.315 |
|
| 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 |
0.447 |
|
| 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 |
0.339 |
|
| 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 |
0.499 |
|
| 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 |
0.41 |
|
| 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 |
0.495 |
|
| 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 |
0.425 |
|
| 2009 |
Mancuso K, Mauck MC, Hauswirth WW, Connor TB, Kuchenbecker JA, Neitz J, Neitz M. Is the high acuity midget ganglion cell system a prerequisite for red-green color vision? Journal of Vision. 9: 46-46. DOI: 10.1167/9.14.46 |
0.333 |
|
| 2009 |
Hauswirth WW, Cideciyan AV, Aleman TS, Kaushal S, Byrne BJ, Schwartz SB, Boye SL, Roman AJ, Pang Jj, Windsor EAM, Sumaroka A, Aguirre GD, Fishman GA, Heon E, Flotte TR, et al. Leber Congenital Amaurosis gene therapy clinical trial Journal of Vision. 9: 27-27. DOI: 10.1167/9.14.27 |
0.338 |
|
| 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 |
0.324 |
|
| 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 |
0.417 |
|
| 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 |
0.502 |
|
| 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 |
0.512 |
|
| 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 |
0.508 |
|
| 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 |
0.527 |
|
| 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 |
0.553 |
|
| 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 |
0.461 |
|
| 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 |
0.409 |
|
| 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 |
0.463 |
|
| 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 |
0.479 |
|
| 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 |
0.782 |
|
| 2008 |
Alexander JJ, Hauswirth WW. Adeno-associated viral vectors and the retina. Advances in Experimental Medicine and Biology. 613: 121-8. PMID 18188936 DOI: 10.1007/978-0-387-74904-4_13 |
0.4 |
|
| 2008 |
Gorbatyuk MS, Hauswirth WW, Lewin AS. Gene therapy for mouse models of ADRP. Advances in Experimental Medicine and Biology. 613: 107-12. PMID 18188934 DOI: 10.1007/978-0-387-74904-4_11 |
0.378 |
|
| 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 |
0.511 |
|
| 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 |
0.502 |
|
| 2008 |
Hauswirth W, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon T, Boye SL, Flotte TR, Byrne B, Jacobson SG. Phase I Trial of Leber Congenital Amaurosis due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results Human Gene Therapy. 81015093227032. DOI: 10.1089/Hgt.2008.107 |
0.395 |
|
| 2008 |
Komáromy AM, Alexander JJ, Cooper AE, Chiodo VA, Glushakova LG, Acland GM, Hauswirth WW, Aguirre GD. Erratum: Targeting gene expression to cones with human cone opsin promoters in recombinant AAV Gene Therapy. 15: 1073-1073. DOI: 10.1038/Gt.2008.107 |
0.446 |
|
| 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 |
0.498 |
|
| 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 |
0.415 |
|
| 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 |
0.392 |
|
| 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 |
0.441 |
|
| 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 |
0.665 |
|
| 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 |
0.46 |
|
| 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 |
0.459 |
|
| 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 |
0.481 |
|
| 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 |
0.338 |
|
| 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 |
0.418 |
|
| 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 |
0.615 |
|
| 2007 |
Mancuso K, Hendrickson AE, Connor TB, Mauck MC, Kinsella JJ, Hauswirth WW, Neitz J, Neitz M. Recombinant adeno-associated virus targets passenger gene expression to cones in primate retina. Journal of the Optical Society of America. a, Optics, Image Science, and Vision. 24: 1411-6. PMID 17429487 DOI: 10.1364/Josaa.24.001411 |
0.495 |
|
| 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 |
0.502 |
|
| 2007 |
Rhee KD, Ruiz A, Duncan JL, Hauswirth WW, Lavail MM, Bok D, Yang XJ. Molecular and cellular alterations induced by sustained expression of ciliary neurotrophic factor in a mouse model of retinitis pigmentosa. Investigative Ophthalmology & Visual Science. 48: 1389-400. PMID 17325188 DOI: 10.1167/Iovs.06-0677 |
0.513 |
|
| 2007 |
Qi X, Sun L, Hauswirth WW, Lewin AS, Guy J. Use of mitochondrial antioxidant defenses for rescue of cells with a leber hereditary optic neuropathy-causing mutation Archives of Ophthalmology. 125: 268-272. PMID 17296905 DOI: 10.1001/Archopht.125.2.268 |
0.353 |
|
| 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 |
0.455 |
|
| 2007 |
Qi X, Lewin AS, Sun L, Hauswirth WW, Guy J. Suppression of mitochondrial oxidative stress provides long-term neuroprotection in experimental optic neuritis Investigative Ophthalmology and Visual Science. 48: 681-691. PMID 17251466 DOI: 10.1167/Iovs.06-0553 |
0.363 |
|
| 2007 |
Qi X, Sun L, Lewin AS, Hauswirth WW, Guy J. The mutant human ND4 subunit of complex I induces optic neuropathy in the mouse Investigative Ophthalmology and Visual Science. 48: 1-10. PMID 17197509 DOI: 10.1167/Iovs.06-0789 |
0.408 |
|
| 2007 |
Gorbatyuk M, Justilien V, Liu J, Hauswirth WW, Lewin AS. Preservation of photoreceptor morphology and function in P23H rats using an allele independent ribozyme Experimental Eye Research. 84: 44-52. PMID 17083931 DOI: 10.1016/J.Exer.2006.08.014 |
0.448 |
|
| 2006 |
Molday LL, Min SH, Seeliger MW, Wu WW, Dinculescu A, Timmers AM, Janssen A, Tonagel F, Hudl K, Weber BH, Hauswirth WW, Molday RS. Disease mechanisms and gene therapy in a mouse model for X-linked retinoschisis. Advances in Experimental Medicine and Biology. 572: 283-9. PMID 17249585 DOI: 10.1007/0-387-32442-9_39 |
0.413 |
|
| 2006 |
Bemelmans AP, Kostic C, Hornfeld D, Jaquet M, Crippa SV, Hauswirth WW, Lem J, Wang Z, Schorderet DE, Munier FL, Wenzel A, Arsenijevic Y. Lentiviral vectors containing a retinal pigment epithelium specific promoter for leber congenital amaurosis gene therapy. Lentiviral gene therapy for LCA. Advances in Experimental Medicine and Biology. 572: 247-53. PMID 17249581 DOI: 10.1007/0-387-32442-9_35 |
0.416 |
|
| 2006 |
Bemelmans AP, Kostic C, Crippa SV, Hauswirth WW, Lem J, Munier FL, Seeliger MW, Wenzel A, Arsenijevic Y. Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis. Plos Medicine. 3: e347. PMID 17032058 DOI: 10.1371/Journal.Pmed.0030347 |
0.533 |
|
| 2006 |
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, ... ... Hauswirth WW, et al. Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. Human Gene Therapy. 17: 845-58. PMID 16942444 DOI: 10.1089/Hum.2006.17.845 |
0.514 |
|
| 2006 |
Haire SE, Pang J, Boye SL, Sokal I, Craft CM, Palczewski K, Hauswirth WW, Semple-Rowland SL. Light-driven cone arrestin translocation in cones of postnatal guanylate cyclase-1 knockout mouse retina treated with AAV-GC1. Investigative Ophthalmology & Visual Science. 47: 3745-53. PMID 16936082 DOI: 10.1167/Iovs.06-0086 |
0.705 |
|
| 2006 |
Glushakova LG, Timmers AM, Pang J, Teusner JT, Hauswirth WW. Human blue-opsin promoter preferentially targets reporter gene expression to rat s-cone photoreceptors. Investigative Ophthalmology & Visual Science. 47: 3505-13. PMID 16877422 DOI: 10.1167/Iovs.05-1670 |
0.467 |
|
| 2006 |
Nusinowitz S, Ridder WH, Pang JJ, Chang B, Noorwez SM, Kaushal S, Hauswirth WW, Heckenlively JR. Cortical visual function in the rd12 mouse model of Leber Congenital Amarousis (LCA) after gene replacement therapy to restore retinal function. Vision Research. 46: 3926-34. PMID 16814838 DOI: 10.1016/J.Visres.2006.05.010 |
0.441 |
|
| 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 |
0.804 |
|
| 2006 |
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, ... ... Hauswirth WW, et al. Safety of recombinant adeno-associated virus type 2-RPE65 vector delivered by ocular subretinal injection. Molecular Therapy : the Journal of the American Society of Gene Therapy. 13: 1074-84. PMID 16644289 DOI: 10.1016/J.Ymthe.2006.03.005 |
0.452 |
|
| 2006 |
Glushakova LG, Timmers AM, Issa TM, Cortez NG, Pang J, Teusner JT, Hauswirth WW. Does recombinant adeno-associated virus-vectored proximal region of mouse rhodopsin promoter support only rod-type specific expression in vivo? Molecular Vision. 12: 298-309. PMID 16617297 |
0.316 |
|
| 2006 |
Sapieha PS, Hauswirth WW, Di Polo A. Extracellular signal-regulated kinases 1/2 are required for adult retinal ganglion cell axon regeneration induced by fibroblast growth factor-2. Journal of Neuroscience Research. 83: 985-95. PMID 16493686 DOI: 10.1002/Jnr.20803 |
0.324 |
|
| 2006 |
Renwick J, Narang MA, Coupland SG, Xuan JY, Baker AN, Brousseau J, Petrin D, Munger R, Leonard BC, Hauswirth WW, Korneluk RG, Tsilfidis C. XIAP-mediated neuroprotection in retinal ischemia. Gene Therapy. 13: 339-47. PMID 16307001 DOI: 10.1038/Sj.Gt.3302683 |
0.48 |
|
| 2006 |
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, ... ... Hauswirth WW, et al. Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis. Molecular Therapy : the Journal of the American Society of Gene Therapy. 13: 565-72. PMID 16223604 DOI: 10.1016/J.Ymthe.2005.09.001 |
0.522 |
|
| 2006 |
Scaria A, Pechan P, Rubin H, Lukason M, Barry E, Ardinger J, Qiu H, Li Q, Peterson J, Hauswirth W, Wadsworth S. 883. AAV-2 Based Gene Therapy Using Novel Anti-VEGF Molecules for Inhibition of Angiogenesis in the Eye Molecular Therapy. 13: S340. DOI: 10.1016/J.Ymthe.2006.08.972 |
0.441 |
|
| 2006 |
Jacobson SG, Acland GM, Aguirre GD, Aleman TS, Boye SL, Schwartz SB, Cideciyan AV, Zeiss CJ, Komaromy AM, Roman AJ, Windsor EA, Sumaroka A, Pearce-Kelling SE, Conlon TJ, Li Q, ... ... Hauswirth WW, et al. 881. Safety, Efficacy and Biodistribution of Recombinant AAV2-RPE65 Vector Delivered by Ocular Subretinal Injection Molecular Therapy. 13: S339. DOI: 10.1016/J.Ymthe.2006.08.970 |
0.429 |
|
| 2006 |
Ye G, Drogemuller C, Thomas D, Scotti M, Kang W, Knop D, Boye S, Pechan P, Peterson J, Hauswirth W, Scaria A, Wadsworth S. 503. A Novel Herpes Simplex Virus Helper Based Production of Adeno- Associated Virus Vectors for Treatment of Retinal Angiogenesis Molecular Therapy. 13: S194. DOI: 10.1016/J.Ymthe.2006.08.573 |
0.459 |
|
| 2006 |
Glushakova LG, Gorbatuyk M, Lu Y, Song S, Zheng M, Shinohara T, Hauswirth WW. 495. Transfer of LEDGF to the Mouse Retina Via Systemic AAV Vector Administration Molecular Therapy. 13: S192. DOI: 10.1016/J.Ymthe.2006.08.565 |
0.435 |
|
| 2006 |
Bemelmans A, Kostic C, Hornfeld D, Tekaya M, Crippa SV, Hauswirth WW, Lem J, Seeliger M, Wenzel A, Arsenijevic Y. 419. Rescue of Cone Photoreceptors after Lentiviral Gene Transfer of Rpe65 cDNA in Knockout Mouse Models of Leber Congenital Amaurosis Molecular Therapy. 13: S161. DOI: 10.1016/J.Ymthe.2006.08.483 |
0.539 |
|
| 2006 |
Dinculescu A, Glushakova L, Min S, Hauswirth W. Adeno-associated virus-vectored gene therapy for retinal disease American Journal of Ophthalmology. 141: 238. DOI: 10.1016/j.ajo.2005.11.028 |
0.395 |
|
| 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 |
0.671 |
|
| 2005 |
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. Pharmacological and rAAV gene therapy rescue of visual functions in a blind mouse model of Leber congenital amaurosis. Plos Medicine. 2: e333. PMID 16250670 DOI: 10.1371/Journal.Pmed.0020333 |
0.461 |
|
| 2005 |
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. 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. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 1072-82. PMID 16226919 DOI: 10.1016/J.Ymthe.2005.08.008 |
0.549 |
|
| 2005 |
Liu J, Timmers AM, Lewin AS, Hauswirth WW. Ribozyme knockdown of the gamma-subunit of rod cGMP phosphodiesterase alters the ERG and retinal morphology in wild-type mice. Investigative Ophthalmology & Visual Science. 46: 3836-44. PMID 16186371 DOI: 10.1167/Iovs.04-1283 |
0.425 |
|
| 2005 |
Gorbatyuk MS, Pang JJ, Thomas J, Hauswirth WW, Lewin AS. Knockdown of wild-type mouse rhodopsin using an AAV vectored ribozyme as part of an RNA replacement approach Molecular Vision. 11: 648-656. PMID 16145542 |
0.37 |
|
| 2005 |
Min SH, Molday LL, Seeliger MW, Dinculescu A, Timmers AM, Janssen A, Tonagel F, Tanimoto N, Weber BH, Molday RS, Hauswirth WW. Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 644-51. PMID 16027044 DOI: 10.1016/J.Ymthe.2005.06.002 |
0.485 |
|
| 2005 |
Loiler SA, Tang Q, Clarke T, Campbell-Thompson ML, Chiodo V, Hauswirth W, Cruz P, Perret-Gentil M, Atkinson MA, Ramiya VK, Flotte TR. Localized gene expression following administration of adeno-associated viral vectors via pancreatic ducts. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 519-27. PMID 15979413 DOI: 10.1016/J.Ymthe.2005.04.017 |
0.412 |
|
| 2005 |
Zhou Y, Pernet V, Hauswirth WW, Di Polo A. Activation of the extracellular signal-regulated kinase 1/2 pathway by AAV gene transfer protects retinal ganglion cells in glaucoma. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 402-12. PMID 15975850 DOI: 10.1016/J.Ymthe.2005.04.004 |
0.466 |
|
| 2005 |
Dinculescu A, Glushakova L, Min SH, Hauswirth WW. Adeno-associated virus-vectored gene therapy for retinal disease. Human Gene Therapy. 16: 649-63. PMID 15960597 DOI: 10.1016/J.Ajo.2005.11.028 |
0.584 |
|
| 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 |
0.554 |
|
| 2005 |
Pernet V, Hauswirth WW, Di Polo A. Extracellular signal-regulated kinase 1/2 mediates survival, but not axon regeneration, of adult injured central nervous system neurons in vivo. Journal of Neurochemistry. 93: 72-83. PMID 15773907 DOI: 10.1111/J.1471-4159.2005.03002.X |
0.316 |
|
| 2005 |
Pang JJ, Chang B, Hawes NL, Hurd RE, Davisson MT, Li J, Noorwez SM, Malhotra R, McDowell JH, Kaushal S, Hauswirth WW, Nusinowitz S, Thompson DA, Heckenlively JR. Retinal degeneration 12 (rd12): a new, spontaneously arising mouse model for human Leber congenital amaurosis (LCA). Molecular Vision. 11: 152-62. PMID 15765048 |
0.342 |
|
| 2005 |
Petrs-Silva H, Chiodo V, Chiarini LB, Hauswirth WW, Linden R. Modulation of the expression of the transcription factor Max in rat retinal ganglion cells by a recombinant adeno-associated viral vector. Brazilian Journal of Medical and Biological Research = Revista Brasileira De Pesquisas MéDicas E BiolóGicas / Sociedade Brasileira De BiofíSica ... [Et Al.]. 38: 375-9. PMID 15761617 DOI: 10.1590/S0100-879X2005000300008 |
0.647 |
|
| 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 |
0.67 |
|
| 2005 |
Gorbatyuk MS, Timmers AM, Jijing P, Hauswirth WW, Lewin AS. 1080. rAAV Delivered Ribozyme Targeting Mouse Opsin to Rescue Vision in P23H Rats Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.627 |
0.409 |
|
| 2005 |
Liu J, Tuli SS, Bloom DC, Schultz GS, Hauswirth WW, Lewin AS. 861. AAV Gene Delivery for the Cornea Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.404 |
0.477 |
|
| 2005 |
Justilien V, Pang J, Hauswirth WW, Lewin AS. 741. AAV Delivered Ribozymes to Recapitulate the Early Stages of Age Related Macular Degeneration Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.281 |
0.417 |
|
| 2005 |
Kostic C, Bemelmans A, Jaquet M, Hornfeld D, Hauswirth WW, Lem J, Wang Z, Schorderet DF, Munier FL, Wenzel A, Arsenijevic Y. 667. Lentiviral Gene Transfer of RPE65 cDNA in Knock-Out Mouse Models of Leber Congenital Amaurosis Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.207 |
0.517 |
|
| 2004 |
Qi X, Lewin AS, Sun L, Hauswirth WW, Guy J. SOD2 gene transfer protects against optic neuropathy induced by deficiency of complex I Annals of Neurology. 56: 182-191. PMID 15293270 DOI: 10.1002/Ana.20175 |
0.389 |
|
| 2004 |
Hennig AK, Ogilvie JM, Ohlemiller KK, Timmers AM, Hauswirth WW, Sands MS. AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 10: 106-16. PMID 15233947 DOI: 10.1016/J.Ymthe.2004.03.018 |
0.522 |
|
| 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 |
0.619 |
|
| 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 DOI: 10.1002/0470092645.Ch14 |
0.837 |
|
| 2004 |
Gorbatyuk M, Pang JJ, Hauswirth WW, Lewin AS. 654. rAAV Mediated Ribozyme Delivery Targeting Mouse Rhodopsin Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.600 |
0.445 |
|
| 2004 |
Qi X, Sun L, Guy J, Hauswirth WW, Lewin AS. 212. AAV-Mediated Gene Transfer Protects Against Mitochondrial Optic Neuropathy in Mice Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.153 |
0.414 |
|
| 2003 |
Petrin D, Baker A, Brousseau J, Coupland S, Liston P, Hauswirth WW, Komeluk RG, Tsilfidis C. XIAP protects photoreceptors from n-methyl-n-nitrosourea-induced retinal degeneration. Advances in Experimental Medicine and Biology. 533: 385-93. PMID 15180289 DOI: 10.1007/978-1-4615-0067-4_49 |
0.409 |
|
| 2003 |
Martin KR, Quigley HA, Zack DJ, Levkovitch-Verbin H, Kielczewski J, Valenta D, Baumrind L, Pease ME, Klein RL, Hauswirth WW. Gene therapy with brain-derived neurotrophic factor as a protection: retinal ganglion cells in a rat glaucoma model. Investigative Ophthalmology & Visual Science. 44: 4357-65. PMID 14507880 DOI: 10.1167/Iovs.02-1332 |
0.449 |
|
| 2003 |
Adamus G, Sugden B, Shiraga S, Timmers AM, Hauswirth WW. Anti-apoptotic effects of CNTF gene transfer on photoreceptor degeneration in experimental antibody-induced retinopathy Journal of Autoimmunity. 21: 121-129. PMID 12935781 DOI: 10.1016/S0896-8411(03)00092-1 |
0.462 |
|
| 2003 |
Petrin D, Baker A, Coupland SG, Liston P, Narang M, Damji K, Leonard B, Chiodo VA, Timmers A, Hauswirth W, Korneluk RG, Tsilfidis C. Structural and functional protection of photoreceptors from MNU-induced retinal degeneration by the X-linked inhibitor of apoptosis. Investigative Ophthalmology & Visual Science. 44: 2757-63. PMID 12766084 DOI: 10.1167/Iovs.02-0729 |
0.396 |
|
| 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 |
0.655 |
|
| 2003 |
Qi X, Lewin AS, Hauswirth WW, Guy J. Optic neuropathy induced by reductions in mitochondrial superoxide dismutase Investigative Ophthalmology and Visual Science. 44: 1088-1096. PMID 12601034 DOI: 10.1167/Iovs.02-0864 |
0.362 |
|
| 2003 |
Qi X, Lewin AS, Hauswirth WW, Guy J. Suppression of complex I gene expression induces optic neuropathy Annals of Neurology. 53: 198-205. PMID 12557286 DOI: 10.1002/Ana.10426 |
0.378 |
|
| 2003 |
Adamus G, Sugden B, Shiraga S, Timmers AM, Hauswirth WW. Erratum to “Anti-apoptotic effects of CNTF gene transfer on photoreceptor degeneration in experimental antibody-induced retinopathy”[Journal of Autoimmunity 21 (2003) 121–129] Journal of Autoimmunity. 21: 393. DOI: 10.1016/J.Jaut.2003.09.008 |
0.358 |
|
| 2002 |
Fritz JJ, Lewin A, Hauswirth W, Agarwal A, Grant M, Shaw L. Development of hammerhead ribozymes to modulate endogenous gene expression for functional studies. Methods (San Diego, Calif.). 28: 276-85. PMID 12413427 DOI: 10.1016/S1046-2023(02)00233-5 |
0.642 |
|
| 2002 |
Zolotukhin S, Potter M, Zolotukhin I, Sakai Y, Loiler S, Fraites TJ, Chiodo VA, Phillipsberg T, Muzyczka N, Hauswirth WW, Flotte TR, Byrne BJ, Snyder RO. Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods (San Diego, Calif.). 28: 158-67. PMID 12413414 DOI: 10.1016/S1046-2023(02)00220-7 |
0.375 |
|
| 2002 |
Guy J, Qi X, Pallotti F, Schon EA, Manfredi G, Carelli V, Martinuzzi A, Hauswirth WW, Lewin AS. Rescue of a mitochondrial deficiency causing Leber Hereditary Optic Neuropathy. Annals of Neurology. 52: 534-42. PMID 12402249 DOI: 10.1002/Ana.10354 |
0.318 |
|
| 2002 |
Bok D, Yasumura D, Matthes MT, Ruiz A, Duncan JL, Chappelow AV, Zolutukhin S, Hauswirth W, LaVail MM. Effects of adeno-associated virus-vectored ciliary neurotrophic factor on retinal structure and function in mice with a P216L rds/peripherin mutation. Experimental Eye Research. 74: 719-35. PMID 12126945 DOI: 10.1006/Exer.2002.1176 |
0.533 |
|
| 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 |
0.83 |
|
| 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 |
0.808 |
|
| 2002 |
McKinnon SJ, Lehman DM, Tahzib NG, Ransom NL, Reitsamer HA, Liston P, LaCasse E, Li Q, Korneluk RG, Hauswirth WW. Baculoviral IAP repeat-containing-4 protects optic nerve axons in a rat glaucoma model. Molecular Therapy : the Journal of the American Society of Gene Therapy. 5: 780-7. PMID 12027563 DOI: 10.1006/Mthe.2002.0608 |
0.501 |
|
| 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 DOI: 10.1016/S0076-6879(02)46066-1 |
0.692 |
|
| 2002 |
Cheng L, Sapieha P, Kittlerová P, Hauswirth WW, Di Polo A. TrkB Gene Transfer Protects Retinal Ganglion Cells from Axotomy-Induced Death In Vivo The Journal of Neuroscience. 22: 3977-3986. DOI: 10.1523/Jneurosci.22-10-03977.2002 |
0.383 |
|
| 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 |
0.567 |
|
| 2001 |
McGee Sanftner LH, Abel H, Hauswirth WW, Flannery JG. Glial cell line derived neurotrophic factor delays photoreceptor degeneration in a transgenic rat model of retinitis pigmentosa. Molecular Therapy : the Journal of the American Society of Gene Therapy. 4: 622-9. PMID 11735347 DOI: 10.1006/Mthe.2001.0498 |
0.518 |
|
| 2001 |
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. Gene therapy restores vision in a canine model of childhood blindness. Nature Genetics. 28: 92-5. PMID 11326284 DOI: 10.1038/Ng0501-92 |
0.534 |
|
| 2001 |
Lewin AS, Hauswirth WW. Ribozyme gene therapy: Applications for molecular medicine Trends in Molecular Medicine. 7: 221-228. PMID 11325634 DOI: 10.1016/S1471-4914(01)01965-7 |
0.374 |
|
| 2001 |
Hauswirth WW, LaVail MM, Flannery JG, Lewin AS. Viral-vectored ribozymes as therapy for autosomal dominant retinal disease Clinical Neuroscience Research. 1: 118-126. DOI: 10.1016/S1566-2772(00)00010-4 |
0.363 |
|
| 2001 |
Hauswirth WW, LaVail MM, Flannery JG, Lewin AS. Viral-vectored ribozymes as therapy for autosomal dominant retinal disease Clinical Neuroscience Research. 1: 118-126. DOI: 10.1016/S1566-2772(00)00010-4 |
0.527 |
|
| 2000 |
Owen R IV, Lewin AP, Peel A, Wang J, Guy J, Hauswirth WW, Stacpoole PW, Flotte TR. Recombinant adeno-associated virus vector-based gene transfer for defects in oxidative metabolism. Human Gene Therapy. 11: 2067-78. PMID 11044909 DOI: 10.1089/104303400750001381 |
0.407 |
|
| 2000 |
Hauswirth WW, Lewin AS. Ribozyme uses in retinal gene therapy Progress in Retinal and Eye Research. 19: 689-710. PMID 11029552 DOI: 10.1016/S1350-9462(00)00007-0 |
0.502 |
|
| 2000 |
LaVail MM, Yasumura D, Matthes MT, Drenser KA, Flannery JG, Lewin AS, Hauswirth WW. Ribozyme rescue of photoreceptor cells in P23H transgenic rats: long-term survival and late-stage therapy. Proceedings of the National Academy of Sciences of the United States of America. 97: 11488-93. PMID 11005848 DOI: 10.1073/Pnas.210319397 |
0.454 |
|
| 2000 |
Hauswirth WW, LaVail MM, Flannery JG, Lewin AS. Ribozyme gene therapy for autosomal dominant retinal disease. Clinical Chemistry and Laboratory Medicine : Cclm / Fescc. 38: 147-53. PMID 10834402 DOI: 10.1515/Cclm.2000.022 |
0.538 |
|
| 2000 |
Shaw LC, Whalen PO, Drenser KA, Yan W, Hauswirth WW, Lewin AS. Ribozymes in treatment of inherited retinal disease. Methods in Enzymology. 316: 761-76. PMID 10800713 DOI: 10.1016/S0076-6879(00)16761-8 |
0.413 |
|
| 2000 |
Hauswirth WW, Lewin AS, Zolotukhin S, Muzyczka N. Production and purification of recombinant adeno-associated virus. Methods in Enzymology. 316: 743-61. PMID 10800712 DOI: 10.1016/S0076-6879(00)16760-6 |
0.334 |
|
| 2000 |
Hauswirth WW, Timmers AM. The eyes have it. Molecular Medicine Today. 6: 51-53. PMID 10681166 DOI: 10.1016/S1357-4310(99)01655-X |
0.432 |
|
| 1999 |
Guy J, Qi X, Wang H, Hauswirth WW. Adenoviral gene therapy with catalase suppresses experimental optic neuritis. Archives of Ophthalmology (Chicago, Ill. : 1960). 117: 1533-9. PMID 10565523 DOI: 10.1001/Archopht.117.11.1533 |
0.338 |
|
| 1999 |
Guy J, Qi X, Muzyczka N, Hauswirth WW. Reporter expression persists 1 year after adeno-associated virus-mediated gene transfer to the optic nerve. Archives of Ophthalmology (Chicago, Ill. : 1960). 117: 929-37. PMID 10408459 DOI: 10.1001/Archopht.117.7.929 |
0.423 |
|
| 1999 |
Owen R, Lewin AS, Wang J, Hauswirth WW, Stacpoole PW, Flotte TR. Adeno-Associated Virus (AAV)-Based Gene Therapy for Defects in Oxidative Metabolism Pediatric Research. 45: 140A-140A. DOI: 10.1203/00006450-199904020-00833 |
0.346 |
|
| 1998 |
Guy J, Qi X, Hauswirth WW. Adeno-associated viral-mediated catalase expression suppresses optic neuritis in experimental allergic encephalomyelitis. Proceedings of the National Academy of Sciences of the United States of America. 95: 13847-52. PMID 9811889 DOI: 10.1073/Pnas.95.23.13847 |
0.35 |
|
| 1998 |
Lewin AS, Drenser KA, Hauswirth WW, Nishikawa S, Yasumura D, Flannery JG, LaVail MM. Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa. Nature Medicine. 4: 967-71. PMID 9701253 DOI: 10.1038/Nm0898-967 |
0.496 |
|
| 1998 |
Lewin A, Drenser K, Hauswirth W, Nishikawa S, Yasumura D, Flannery J, LaVail M. Erratum: Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa Nature Medicine. 4: 1081-1081. DOI: 10.1038/2077 |
0.329 |
|
| 1997 |
Flannery JG, Zolotukhin S, Vaquero MI, LaVail MM, Muzyczka N, Hauswirth WW. Efficient photoreceptor-targeted gene expression in vivo by recombinant adeno-associated virus. Proceedings of the National Academy of Sciences of the United States of America. 94: 6916-21. PMID 9192666 DOI: 10.1073/pnas.94.13.6916 |
0.472 |
|
| 1996 |
Zolotukhin S, Potter M, Hauswirth WW, Guy J, Muzyczka N. A "Humanized" green fluorescent protein cDNA adapted for high-level expression in mammalian cells Journal of Virology. 70: 4646-4654. PMID 8676491 DOI: 10.1128/Jvi.70.7.4646-4654.1996 |
0.439 |
|
| 1996 |
Muise RC, Hauswirth WW. Selective DNA amplification regulates transcript levels in plant mitochondria. Current Genetics. 28: 113-21. PMID 8590461 DOI: 10.1007/Bf00315776 |
0.308 |
|
| 1996 |
van Ginkel PR, Timmers AM, Szél A, Hauswirth WW. Topographical regulation of cone and rod opsin genes: parallel, position dependent levels of transcription. Brain Research. Developmental Brain Research. 89: 146-9. PMID 8575088 DOI: 10.1016/0165-3806(95)00122-T |
0.411 |
|
| 1995 |
DesJardin LE, Lockwood MK, Hauswirth WW. Bovine opsin gene expression exhibits a late fetal to adult regulatory switch. Journal of Neuroscience Research. 40: 728-36. PMID 7543158 DOI: 10.1002/Jnr.490400604 |
0.407 |
|
| 1993 |
Timmers AM, Newton BR, Hauswirth WW. Synthesis and stability of retinal photoreceptor mRNAs are coordinately regulated during bovine fetal development. Experimental Eye Research. 56: 257-65. PMID 8386100 DOI: 10.1006/Exer.1993.1034 |
0.361 |
|
| 1993 |
Flannery JG, Dinehart WJ, Street C, Hauswirth WW. In Situ Hybridization of Retinal Messenger RNA Using Nonradioactive Synthetic Probes Methods in Neurosciences. 15: 357-376. DOI: 10.1016/B978-0-12-185279-5.50032-7 |
0.397 |
|
| 1992 |
Hauswirth WW, Langerijt AVD, Timmers AM, Adamus G, Ulshafer RJ. Early expression and localization of rhodopsin and interphotoreceptor retinoid-binding protein (IRBP) in the developing fetal bovine retina. Experimental Eye Research. 54: 661-670. PMID 1623951 DOI: 10.1016/0014-4835(92)90021-J |
0.364 |
|
| 1991 |
Lawlor DA, Dickel CD, Hauswirth WW, Parham P. Ancient HLA genes from 7,500-year-old archaeological remains. Nature. 349: 785-8. PMID 2000147 DOI: 10.1038/349785A0 |
0.347 |
|
| 1986 |
Tanhauser SM, Hauswirth WW, Laipis PJ. Conserved restriction sites within the ribosomal RNA genes of vertebrates. Biochimica Et Biophysica Acta. 866: 19-25. PMID 3004584 DOI: 10.1016/0167-4781(86)90095-3 |
0.325 |
|
| 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 DOI: 10.1128/Jvi.33.2.739-748.1980 |
0.589 |
|
| 1979 |
Berns KI, Hauswirth WW. Adeno-associated viruses. Advances in Virus Research. 25: 407-49. PMID 230727 DOI: 10.1016/S0065-3527(08)60574-6 |
0.6 |
|
| 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 DOI: 10.1101/Sqb.1979.043.01.085 |
0.583 |
|
| 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 |
0.585 |
|
| 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 |
0.529 |
|
| 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 |
0.538 |
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