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Article

Synergistic effects of central nervous system‐directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis

1 Feb 2012

Abstract excerpt

OBJECTIVE: Infantile neuronal ceroid lipofuscinosis (INCL) is an inherited childhood neurodegenerative disorder caused by the loss of palmitoyl protein thioesterase-1 (PPT1) activity. Affected children suffer from blindness, epilepsy, motor dysfunction, cognitive decline, and premature death. The Ppt1(-/-) mouse shares the histological and clinical features of INCL. Previous single-therapy approaches using small...

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PMID: 22368049
DOI: 10.1002/ana.23545

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