Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease.

Published 2021 View Full Article

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Title
Waning efficacy in a long-term AAV-mediated gene therapy study in the murine model of Krabbe disease.
Authors
Keywords
globoid cell leukodystrophy, AAV9, gene therapy, psychosine, multi-focal sclerosis, microglia, lysosomal dysfunction, demyelination, fibrinogen, tight junction
Journal
MOLECULAR THERAPY
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2021-01-26
DOI
10.1016/j.ymthe.2021.01.026

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