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Article

Combination small molecule PPT1 mimetic and CNS‐directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis

6 Feb 2012

Abstract excerpt

Infantile neuronal ceroid lipofuscinosis (INCL) is a profoundly neurodegenerative disease of children caused by a deficiency in the lysosomal enzyme palmitoyl protein thioesterase-1 (PPT1). There is currently no effective therapy for this invariably fatal disease. To date, preclinical experiments using single treatments have resulted in incremental clinical improvements. Therefore, we determined the efficacy of...

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PMID: 22310926
DOI: 10.1007/s10545-011-9446-x

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