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T.I.2 Exon skipping and PRO044 in Duchenne muscular dystrophy: Extending the program

✍ Scribed by Ferlini, A.; Goemans, N.; Tulinius, M.; Niks, E.H.; Dorricott, S.; Morgan, A.; Lourbakos, A.; Kimpe, S.; Wilson, R.; Armaroli, A.; van Deutekom, J.; Campion, G.

Book ID
121390600
Publisher
Elsevier Science
Year
2013
Tongue
English
Weight
48 KB
Volume
23
Category
Article
ISSN
0960-8966

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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