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Full-length dystrophin gene transfer to the mdx mouse in utero

✍ Scribed by Reay, D P; Bilbao, R; Koppanati, B M; Cai, L; O'Day, T L; Jiang, Z; Zheng, H; Watchko, J F; Clemens, P R


Book ID
109855187
Publisher
Nature Publishing Group
Year
2008
Tongue
English
Weight
173 KB
Volume
15
Category
Article
ISSN
0969-7128

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## Abstract ## Background Brain‐directed prenatal gene therapy may benefit some lysosomal storage diseases that affect the central nervous system (CNS) before birth. Our previous study showed that intrauterine introduction of recombinant adenoviruses into cerebral ventricles results in efficient g