Gene therapy for juvenile Batten's disease

摘要

PROBLEM TO BE SOLVED: To provide gene therapy for juvenile Batten's disease. Kind Code: A1 Compositions and methods for the treatment of juvenile neuronal ceroid lipofuscinosis (JNCL), also known as juvenile Batten's disease. In certain embodiments, the composition comprises, but is not limited to, an adeno-associated virus (AAV) construct, including a self-complementary adeno-associated virus (sc-AAV) construct expressing the human gene CLN3 (or CLN3 cDNA). Not done. The construct may be a recombinant vector comprising a recombinant adeno-associated virus (AAV) genome or derivative thereof and a promoter operably linked to a polynucleotide sequence encoding CLN3 or its equivalent. [Selection diagram] None