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T.P.4.02 Evaluation of novel compounds for upregulation of utrophin in animal models of Duchenne muscular dystrophy therapy

✍ Scribed by R.J. Fairclough; A. Potter; D. Powell; S. Squire; M. Bland; A. Bareja; J. Tinsley; K.E. Davies


Book ID
116793713
Publisher
Elsevier Science
Year
2008
Tongue
English
Weight
48 KB
Volume
18
Category
Article
ISSN
0960-8966

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## Abstract ## Background Targeted splice modulation of pre‐mRNA transcripts by antisense oligonucleotides (AOs) can correct the function of aberrant disease‐related genes. Duchenne muscular dystrophy (DMD) arises as a result of mutations that interrupt the open‐reading frame in the DMD gene encod