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

Title
Combination small molecule PPT1 mimetic and CNS-directed gene therapy as a treatment for infantile neuronal ceroid lipofuscinosis
Authors
Keywords
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Journal
JOURNAL OF INHERITED METABOLIC DISEASE
Volume 35, Issue 5, Pages 847-857
Publisher
Springer Nature
Online
2012-02-06
DOI
10.1007/s10545-011-9446-x

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