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

@inproceedings{Sun2010GeneTW,
  title={Gene Therapy with a Promoter Targeting Both Rods and Cones Rescues Retinal Degeneration Caused by AIPL1 Mutations},
  author={Xun Sun and Basil S. Pawlyk and Xiaoyun Xu and Xiaoqing Liu and Oleg V Bulgakov and Michael Adamian and Michael A. Sandberg and Shahrokh C. Khani and Mei-Hong Tan and Alexander D. Smith and Robin R Ali and Tiansen Li},
  booktitle={Gene Therapy},
  year={2010}
}
Aryl hydrocarbon receptor-interacting protein-like 1 (AIPL1) is required for the biosynthesis of photoreceptor phosphodiesterase (PDE). Gene defects in AIPL1 cause a heterogeneous set of conditions ranging from Leber's congenital amaurosis (LCA), the severest form of early-onset retinal degeneration, to milder forms such as retinitis pigmentosa (RP) and cone-rod dystrophy. In mice, null and hypomorphic alleles cause retinal degeneration similar to human LCA and RP, respectively. Thus these… CONTINUE READING

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