Article
Subretinal delivery of AAV5-mediated human Pde6b gene ameliorates the disease phenotype in a rat model of retinitis pigmentosa.
Molecular vision - 1 Jan 2025
Kim Hee Jong, Kwak Ji Hoon, Choi Jun Sub, Kim Jin, Moon Seo Yun, Lee Steven Hyun Seung, Lee Heuiran, Park Keerang, Lee Joo Yong, Won So-Yoon
Abstract excerpt
Purpose: A genetic disorder that affects the beta subunit of cyclic guanosine monophosphate-phosphodiesterase type 6 (PDE6B) in humans leads to autosomal recessive retinitis pigmentosa (RP). This condition causes severe vision loss in early life due to fast deterioration of photoreceptors. This study evaluated the therapeutic potential of subretinal delivery of the adeno-associated virus (AAV)5-mediated human...
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