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P3.02 AAV-U7snRNA mediated multi exon-skipping for Duchenne muscular dystrophy

โœ Scribed by A. Goyenvalle; A. Babbs; L. Garcia; K.E. Davies


Book ID
116794574
Publisher
Elsevier Science
Year
2010
Tongue
English
Weight
40 KB
Volume
20
Category
Article
ISSN
0960-8966

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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