Retinitis Pigmentosa Gene Therapy
Recent studies from Harvard University add new data to retinitis pigmentosa gene therapy
Published in Gene Therapy Weekly, January 21st, 2010
Scientists discuss in 'Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations' new findings in retinitis pigmentosa. According to recent research published in the journal Gene Therapy, "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...
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Source: Gene Therapy Weekly (2010-01-21)
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