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Gene therapy of MLD model mice by intrathecal and intravenous injection of AAV9 vector

✍ Scribed by Miyake, Noriko; Miyake, Koichi; Shimada, Takashi


Book ID
122300966
Publisher
Elsevier Science
Year
2014
Tongue
English
Weight
65 KB
Volume
111
Category
Article
ISSN
1096-7192

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## Abstract ## Background The success of gene therapy for inherited neurodegenerative diseases such as metachromatic leukodystrophy (MLD) depends on the development of efficient gene delivery throughout the brain guarded by the blood–brain barrier and achieves distribution of the deficient enzyme