abstract |
Viral vector compositions are described comprising polynucleotide sequences that express one or more biologically active mammalian guanylate cyclase proteins; methods are also described for use in the prevention, treatment and / or relief of at least one or more symptoms of a disease, disorder, abnormal condition or dysfunction, which result, at least in part, from a guanylate cyclase deficiency in vivo; in particular embodiments, the use of recombinant adeno-associated viral vectors (rAAV) to treat or alleviate the symptoms of Leber's congenital amaurosis, and also other conditions caused by an absence or reduction of the expression of a specific retinal guanylate cyclase 1 (retGC1) functional. |