Intrathecal and Intravenous Combination Gene Therapy for the Treatment of Infant Batten's Disease

摘要

A method of treating IBD or an IBD-associated disorder in a subject in need thereof, comprising the steps of combining intrathecal administration of a polynucleotide comprising a CLN1 open reading frame with intravenous administration of said polynucleotide. method to include. The polynucleotide comprising the CLN1 open reading frame is a wild-type CLN1 polynucleotide. In another embodiment, a polynucleotide comprising the CLN1 open reading frame comprises a codon optimized polynucleotide sequence of the polynucleotide or a complement thereof and is codon optimized for expression in a human cell.