Title : CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis.

Pub. Date : 2006 Mar

PMID : 16364693






1 Functional Relationships(s)
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1 The AAV-treated animals had localized increases in PPT1 activity, decreased autofluorescent material, improved histologic parameters, and increased brain mass. CHEMBL2031461 palmitoyl-protein thioesterase 1 Homo sapiens