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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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| Main Authors: | , , , , , , , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2009
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| Assuntos: | |
| Acesso em linha: | 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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