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T.O. 1 Antisense-induced exon skipping in Duchenne muscular dystrophy patients

โœ Scribed by A.A.M. Janson; I.H.B. Ginjaar; A. Aartsma-Rus; G.-J.B. van Ommen; A. van der Kooi; S. de Kimpe; P. Ekhart; G. Platenburg; J.J.G.M. Verschuuren; J.C.T. van Deutekom

Book ID
116792701
Publisher
Elsevier Science
Year
2006
Tongue
English
Weight
45 KB
Volume
16
Category
Article
ISSN
0960-8966

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Theoretic applicability of antisense-med
Theoretic applicability of antisense-mediated exon skipping for Duchenne muscular dystrophy mutations
โœ Annemieke Aartsma-Rus; Ivo Fokkema; Jan Verschuuren; Ieke Ginjaar; Judith van De ๐Ÿ“‚ Article ๐Ÿ“… 2009 ๐Ÿ› John Wiley and Sons ๐ŸŒ English โš– 176 KB

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