Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes

Alessandro Fraldi, Kim Hemsley, Allison Crawley, Alessia Lombardi, Adeline Lau, Leanne Sutherland, Alberto Auricchio, Andrea Ballabio, John J. Hopwood

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Neuroscience

Biochemistry, Genetics and Molecular Biology