abstract |
The present invention provides rAAV-guanylate cyclase compositions and methods for treating Leber's congenital black cataract 1 (LCA1). Disclosed is a viral vector composition comprising a polynucleotide sequence that expresses one or more biologically active mammalian guanylate cyclase proteins. 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 guanylate cyclase deficiency in vivo The method of is also disclosed. In a specific embodiment, a recombinant for treating or ameliorating the symptoms of Leber congenital black cataract and other conditions caused by the absence or reduction in expression of functional retina-specific guanylate cyclase 1 (retGC1) The use of adeno-associated virus (rAAV) vectors is disclosed. 【Selection chart】 None |