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T.P.2.04 Optimization of antisense-mediated exon skipping in mouse models for Duchenne muscular dystrophy

✍ Scribed by A. Aartsma-Rus; H.A. Heemskerk; C.L. de Winter; M. van Putten; S. De Kimpe; J.C.T. van Deutekom; G.J.B. van Ommen

Book ID: 116793532
Publisher: Elsevier Science
Year: 2008
Tongue: English
Weight: 49 KB
Volume: 18
Category: Article
ISSN: 0960-8966
DOI: 10.1016/j.nmd.2008.06.116

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## Abstract ## Background Antisense‐mediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allowing generation of partially functional dystrophin and conversion of a severe Duchenne