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Exon-skipping therapy for Duchenne muscular dystrophy

✍ Scribed by Akinori Nakamura; Shin'ichi Takeda

Book ID
108958161
Publisher
John Wiley and Sons
Year
2009
Tongue
English
Weight
127 KB
Volume
29
Category
Article
ISSN
0919-6544

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## Exon skipping to change Duchenne into Becker dystrophy The exon-skipping technique tries to change a Duchenne into a Becker mutation. If a deletion or a point mutation disturbs the reading frame, and thus causes Duchenne dystrophy, the reading frame can be restored by removing one or more exons

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## Abstract Mutations in the dystrophin gene cause an X‐linked genetic disorder: Duchenne muscular dystrophy (DMD). Stem cell therapy is an attractive method to treat DMD because a small number of cells are required to obtain a therapeutic effect. Here, we discussed about multiple types of myogenic