Article
Gene Therapy Rescues Retinal Degeneration in Receptor Expression-Enhancing Protein 6 Mutant Mice.
Human gene therapy - 1 Mar 2019
Zaneveld Smriti Agrawal, Eblimit Aiden, Liang Qingnan, Bertrand Renae, Wu Nathaniel, Liu Hehe, Nguyen Quynh, Zaneveld Jacques, Wang Keqing, Li Yumei, Chen Rui
Abstract excerpt
Hereditary retinal dystrophy is clinically defined as a broad group of chronic and progressive disorders that affect visual function by causing photoreceptor degeneration. Previously, we identified mutations in the gene encoding receptor expression-enhancing protein 6 (REEP6), in individuals with autosomal recessive retinitis pigmentosa (RP), the most common form of inherited retinal dystrophy. One individual was...
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