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127 results

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Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.
Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG. Hauswirth WW, et al. Among authors: boye sl. Hum Gene Ther. 2008 Oct;19(10):979-90. doi: 10.1089/hum.2008.107. Hum Gene Ther. 2008. PMID: 18774912 Free PMC article. Clinical Trial.
Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.
Pang JJ, Dai X, Boye SE, Barone I, Boye SL, Mao S, Everhart D, Dinculescu A, Liu L, Umino Y, Lei B, Chang B, Barlow R, Strettoi E, Hauswirth WW. Pang JJ, et al. Among authors: boye sl, boye se. Mol Ther. 2011 Feb;19(2):234-42. doi: 10.1038/mt.2010.273. Epub 2010 Dec 7. Mol Ther. 2011. PMID: 21139570 Free PMC article.
Virally delivered channelrhodopsin-2 safely and effectively restores visual function in multiple mouse models of blindness.
Doroudchi MM, Greenberg KP, Liu J, Silka KA, Boyden ES, Lockridge JA, Arman AC, Janani R, Boye SE, Boye SL, Gordon GM, Matteo BC, Sampath AP, Hauswirth WW, Horsager A. Doroudchi MM, et al. Among authors: boye sl, boye se. Mol Ther. 2011 Jul;19(7):1220-9. doi: 10.1038/mt.2011.69. Epub 2011 Apr 19. Mol Ther. 2011. PMID: 21505421 Free PMC article.
Gene therapy for retinitis pigmentosa caused by MFRP mutations: human phenotype and preliminary proof of concept.
Dinculescu A, Estreicher J, Zenteno JC, Aleman TS, Schwartz SB, Huang WC, Roman AJ, Sumaroka A, Li Q, Deng WT, Min SH, Chiodo VA, Neeley A, Liu X, Shu X, Matias-Florentino M, Buentello-Volante B, Boye SL, Cideciyan AV, Hauswirth WW, Jacobson SG. Dinculescu A, et al. Among authors: boye sl. Hum Gene Ther. 2012 Apr;23(4):367-76. doi: 10.1089/hum.2011.169. Epub 2012 Jan 26. Hum Gene Ther. 2012. PMID: 22142163 Free PMC article.
127 results