Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes.
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A. Ballabio | A. Fraldi | J. Hopwood | K. Hemsley | A. Auricchio | A. Lombardi | A. Crawley | Adeline A. Lau | Leanne Sutherland | L. Sutherland