AAV-Mediated Gene Therapy for Glycosphingolipid Biosynthesis Deficiencies
Author:
Funder
University of Massachusetts Medical School
Publisher
Elsevier BV
Subject
Molecular Biology,Molecular Medicine
Reference15 articles.
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3. Overexpression of the wild-type SPT1 subunit lowers desoxysphingolipid levels and rescues the phenotype of HSAN1;Eichler;J. Neurosci.,2009
4. Infantile-onset symptomatic epilepsy syndrome caused by a homozygous loss-of-function mutation of GM3 synthase;Simpson;Nat. Genet.,2004
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