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Antisense-Induced Multiexon Skipping for Duchenne Muscular Dystrophy Makes More Sense

✍ Scribed by Annemieke Aartsma-Rus; Anneke A.M. Janson; Wendy E. Kaman; Mattie Bremmer-Bout; Gert-Jan B. van Ommen; Johan T. den Dunnen; Judith C.T. van Deutekom


Book ID
117854358
Publisher
American Society of Human Genetics
Year
2004
Tongue
English
Weight
620 KB
Volume
74
Category
Article
ISSN
0002-9297

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Antisense-mediated exon skipping aiming for reading frame restoration is currently a promising therapeutic application for Duchenne muscular dystrophy (DMD). This approach is mutation specific, but as the majority of DMD patients have deletions that cluster in hotspot regions, the skipping of a smal