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

标题
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
作者
关键词
AAVrh10, GALC, Galactocerebrosidase, Gene therapy, Globoid cell leukodystrophy, Krabbe disease
出版物
MOLECULAR GENETICS AND METABOLISM
Volume 114, Issue 3, Pages 459-466
出版商
Elsevier BV
发表日期
2014-12-12
DOI
10.1016/j.ymgme.2014.12.300

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