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Eyal Banin,Ayala Ejzenberg,Lina Zelinger, Edward Averbukh - Ophthalmology, Hadassah-Hebrew Univ Med Ctr, Jerusalem, Israel
Raaya Ezra-Elia, Ron Ofri  -Koret School of Veterinary Medicine, Hebrew University of Jerusalem, Rehovot, Israel
William W Hauswirth - Ophthalmology, University of Florida, Gainsville, FL
 
 
 

Purpose: Mutations in the CNGA3 gene cause achromatopsia in humans. We identified a spontaneously-arising sheep model for this disease (Shamir et. al., Vet J. 185(2):130-7, 2010; Reicher et al., Genomics 95:101-4, 2010), and used this model to test the efficacy and safety of viral vector-mediated gene therapy.

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Gene therapy in a sheep model of CNGA3 achromatopsia
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Eyal Banin,Ayala Ejzenberg,Lina Zelinger, Edward Averbukh - Ophthalmology, Hadassah-Hebrew Univ Med Ctr, Jerusalem, Israel
Raaya Ezra-Elia, Ron Ofri  -Koret School of Veterinary Medicine, Hebrew University of Jerusalem, Rehovot, Israel
William W Hauswirth - Ophthalmology, University of Florida, Gainsville, FL
 
 
 
Gene therapy in a sheep model of CNGA3 achromatopsia .

Purpose: Mutations in the CNGA3 gene cause achromatopsia in humans. We identified a spontaneously-arising sheep model for this disease (Shamir et. al., Vet J. 185(2):130-7, 2010; Reicher et al., Genomics 95:101-4, 2010), and used this model to test the efficacy and safety of viral vector-mediated gene therapy.

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