An improved, novel, systemically administered AAV gene therapy for treatment of CLN3 juvenile neuronal ceroid lipofuscinosis
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DOI: 10.1016/j.ymgme.2018.12.202
Abstract: Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) is a lysosomal storage disease caused by an autosomal recessively inherited mutation in the CLN3 gene. JNCL is a progressive neurodegenerative disorder in which the central nervous system (CNS) is… read more here.
Keywords: neuronal ceroid; juvenile neuronal; ceroid lipofuscinosis; biodistribution ... See more keywords