Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features

Title
Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features
Authors
Keywords
AAVrh10, GALC, Galactocerebrosidase, Gene therapy, Globoid cell leukodystrophy, Krabbe disease
Journal
MOLECULAR GENETICS AND METABOLISM
Volume 114, Issue 3, Pages 459-466
Publisher
Elsevier BV
Online
2014-12-12
DOI
10.1016/j.ymgme.2014.12.300

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