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Gene Augmentation Therapy for a Missense Substitution in the cGMP-Binding Domain of Ovine CNGA3 Gene Restores Vision in Day-Blind Sheep.
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. Gootwine E, et al. Among authors: rosov a. Invest Ophthalmol Vis Sci. 2017 Mar 1;58(3):1577-1584. doi: 10.1167/iovs.16-20986. Invest Ophthalmol Vis Sci. 2017. PMID: 28282490 Free PMC article.
Safety and Efficacy Evaluation of rAAV2tYF-PR1.7-hCNGA3 Vector Delivered by Subretinal Injection in CNGA3 Mutant Achromatopsia Sheep.
Gootwine E, Ofri R, Banin E, Obolensky A, Averbukh E, Ezra-Elia R, Ross M, Honig H, Rosov A, Yamin E, Ye GJ, Knop DR, Robinson PM, Chulay JD, Shearman MS. Gootwine E, et al. Among authors: rosov a. Hum Gene Ther Clin Dev. 2017 Jun;28(2):96-107. doi: 10.1089/humc.2017.028. Epub 2017 May 5. Hum Gene Ther Clin Dev. 2017. PMID: 28478700
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