recombinant adeno-associated viral vector, plurality of infectious viral particles, mammalian host cells, kit, use of composition, methods for preventing, treating or ameliorating disease, dysfunction, disorder, deficiency or abnormal condition in one or more symptoms thereof to provide therapeutically effective amount of guanylate cyclase peptide, polypeptide or protein to the biologically active mammal in need thereof, to increase the level of biologically retg1 protein in one or more mammalian retinal cells and to treat or ameliorate one or more symptoms of retinal dystrophy in mammal

摘要

recombinant adeno-associated viral vector, plurality of infectious viral particles, mammalian host cells, kit, use of composition, methods for preventing, treating or ameliorating disease, dysfunction, disorder, deficiency or abnormal condition in one or more symptoms thereof to provide therapeutically effective amount of guanylate cyclase peptide, polypeptide or protein to the biologically active mammal in need thereof, to increase the level of biologically retg1 protein in one or more mammalian retinal cells and to treat or ameliorate one or more symptoms of Mammalian retinal dystrophy vector compositions comprising polynucleotide sequences expressing one or more biologically active mammalian guanylate kinase proteins are described. Methods for its use in the prevention, treatment and / or amelioration of at least one or more symptoms of a disease, disorder, abnormal condition, or dysfunction resulting at least in part from guanylate cyclase deficiency in vivo are also described. in particular embodiments, the use of recombinant adeno-associated viral vectors (RAAVs) to treat or ameliorate the symptoms of congenital leber amaurosis, as well as other conditions caused by the absence or reduction in expression of a functional retinal guanylate cyclase 1 ( retgc1).