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rAAV-Guanylate Cyclase Compositions and Methods for Treating Lebers Congenital Amaurosis-1 (LCA1)
rAAV-Guanylate Cyclase Compositions and Methods for Treating Lebers Congenital Amaurosis-1 (LCA1)
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机译:rAAV-瓜南酸酯环化酶组成和方法治疗Lebers先天性阿马育病1(LCA1)
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摘要
Disclosed are viral vector compositions comprising polynucleotide sequences that express one or more biologically-active mammalian guanylate cyclase proteins. Also disclosed are methods for their use in preventing, treating, and/or ameliorating at least one or more symptoms of a disease, disorder, abnormal condition, or dysfunction resulting at least in part from a guanylate cyclase deficiency in vivo. In particular embodiments, the use of recombinant adeno-associated viral (rAAV) vectors to treat or ameliorate symptoms of Leber's congenital amaurosis, as well as other conditions caused by an absence or reduction in the expression of a functional retinal-specific guanylate cyclase 1 (retGC1).
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