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The Hopes and Fears of In Utero Gene Therapy for Genetic Disease—A Review

✍ Scribed by C. Coutelle; M. Themis; S. Waddington; L. Gregory; M. Nivsarkar; S. Buckley; T. Cook; C. Rodeck; D. Peebles; A. David

Book ID
114115837
Publisher
Elsevier Science
Year
2003
Tongue
English
Weight
106 KB
Volume
24
Category
Article
ISSN
0143-4004

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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