Article
Tyrosine-mutant AAV8 delivery of human MERTK provides long-term retinal preservation in RCS rats.
Investigative ophthalmology & visual science - 6 Apr 2012
Deng Wen-Tao, Dinculescu Astra, Li Qiuhong, Boye Sanford L, Li Jie, Gorbatyuk Marina S, Pang Jijing, Chiodo Vince A, Matthes Michael T, Yasumura Douglas, Liu Li, Alkuraya Fowzan S, Zhang Kang, Vollrath Douglas, LaVail Matthew M, Hauswirth William W
Abstract excerpt
PURPOSE: The absence of Mertk in RCS rats results in defective RPE phagocytosis, accumulation of outer segment (OS) debris in the subretinal space, and subsequent death of photoreceptors. Previous research utilizing Mertk gene replacement therapy in RCS rats provided proof of concept for treatment of this form of recessive retinitis pigmentosa (RP); however, the beneficial effects on retinal function were...
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