RECOMBINANT ADENO-ASSOCIATED VIRAL (rAAV) VECTOR ENCODING BIOLOGICALLY ACTIVE RETINA-SPECIFIC MAMMALIAN GUANYLATE CYCLASE, CAPABLE OF PRESERVING OR RESTORING CONE-MEDIATED FUNCTION IN THE EYE OF A MAMMAL

摘要

The invention relates to a recombinant adeno-associated viral (rAAV) vector encoding biologically-active retina-specific mammalian guanylate cyclase, that is capable of preserving or restoring cone-mediated function in the eye of the mammal. The invention also relates to a corresponding composition comprising the claimed rAAV vector. The claimed vector and composition are used in a method for preventing, treating or ameliorating a disease, disorder, dysfunction or abnormal condition of a mammalian eye; in a method for increasing the level of biologically-active retGCl protein in retinal cells of a mammal; in a method for treating or ameliorating symptoms of retinal dystrophy in a mammal. In particular embodiment, the invention is directed against Leber's congenital amaurosis-1 (LCA1) in a mammal.