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Efficient gene transfer to muscle satellite cells by in utero intravenous delivery of adeno-associated viral vector

✍ Scribed by David H. Stitelman; Archana Bora; Traas Jeremy; Demetri Merianos; Alan W. Flake; Tim Brazelton


Book ID
113679486
Publisher
Elsevier Science
Year
2009
Tongue
English
Weight
97 KB
Volume
209
Category
Article
ISSN
1072-7515

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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