Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

Defects in the photoreceptor-specific gene encoding aryl hydrocarbon receptor-interacting protein-like 1 (AIPL1) are clinically heterogeneous and present as Leber Congenital Amaurosis, the severest form of early-onset retinal dystrophy and milder forms of retinal dystrophies such as juvenile retinit...

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Hlavní autoři: Tan, Mei Hong, Smith, Alexander J., Pawlyk, Basil, Xu, Xiaoyun, Liu, Xiaoqing, Bainbridge, James B., Basche, Mark, McIntosh, Jenny, Tran, Hoai Viet, Nathwani, Amit, Li, Tiansen, Ali, Robin R.
Médium: Artigo
Jazyk:Inglês
Vydáno: Oxford University Press 2009
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC2722233/
https://ncbi.nlm.nih.gov/pubmed/19299492
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddp133
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