-1LCA1 - - RAAV-GUANYLATE CYCLASE COMPOSITIONS AND METHODS FOR TREATING LEBER'S CONGENITAL AMAUROSIS-1 LCA1

摘要

A viral vector composition comprising a polynucleotide sequence expressing one or more biologically active mammalian guanylate cyclase proteins. Also disclosed is a method of its use in preventing, treating and / or ameliorating one or more symptoms of a disease, disorder, condition or dysfunction that results, at least in part, from a lack of guanilate cyclase in vivo. In certain embodiments, recombinant adeno-associated to treat or ameliorate the symptoms of Lever congenital black intestine as well as other diseases caused by the absence or reduction of expression of functional retinal-specific guanylate cyclase 1 (retGC1). Use of viral (rAAV) vectors is disclosed.