Gene Therapy with a Promoter Targeting Both Rods and Cones Rescues Retinal Degeneration Caused by AIPL1 Mutations

AIPL1 is required for the biosynthesis of photoreceptor phosphodiesterase (PDE)1–3. Gene defects in AIPL1 cause a heterogeneous set of conditions ranging from Leber Congenital Amaurosis (LCA), the severest form of early-onset retinal degeneration, to milder forms such as retinitis pigmentosa (RP) an...

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Hlavní autoři: Sun, Xun, Pawlyk, Basil, Xu, Xiaoyun, Liu, Xiaoqing, Bulgakov, Oleg, Adamian, Michael, Sandberg, Michael A., Khani, Shahrokh C., Tan, Mei-Hong, Smith, Alexander J., Ali, Robin R., Li, Tiansen
Médium: Artigo
Jazyk:Inglês
Vydáno: 2009
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2804971/
https://ncbi.nlm.nih.gov/pubmed/19710705
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2009.104
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