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

Research output: Contribution to journal › Article › peer-review

105 Citations (Scopus)

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Biochemistry, Genetics and Molecular Biology

Pharmacology, Toxicology and Pharmaceutical Science

Neuroscience