Safety and Efficacy of AAV2tYF-PR1.7-CNGA3 in CNGA3-deficient Sheep

Mark S. Shearman, Elisha Gootwine, Eyal Banin, Ron Ofri, Raaya Ezra-Elia, Guo-jie Ye, Paulette Robinson, David Knop, Jeffrey D. Chulay

Research output: Contribution to journalMeeting Abstractpeer-review


This is an abstract that was submitted for the 2017 ARVO Annual Meeting, held in Baltimore, MD, May 7-11, 2017.
Purpose : Achromatopsia is an inherited retinal disorder characterized by markedly reduced visual acuity, extreme light sensitivity and absence of color discrimination. AGTC is developing AAV2tYF-PR1.7-CNGA3, a recombinant adeno-associated virus (rAAV) vector expressing CNGA3, for treatment of CNGA3-related achromatopsia. Here we report results of a toxicology and efficacy study of this vector administered by subretinal injection in CNGA3-deficient sheep.
Original languageEnglish
JournalInvestigative Ophthalmology and Visual Science
Issue number8
StatePublished - Jun 2017


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