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A Prospective Study in the Rational Design of Efficient Antisense Oligonucleotides for Exon Skipping in the DMD Gene

✍ Scribed by Pramono, Zacharias Aloysius Dwi; Wee, Keng Boon; Wang, Jian Li; Chen, Yi Jun; Xiong, Qian Bin; Lai, Poh San; Yee, Woon Chee

Book ID
120034390
Publisher
Mary Ann Liebert
Year
2012
Tongue
English
Weight
638 KB
Volume
23
Category
Article
ISSN
1043-0342

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## Abstract ## Background Duchenne muscular dystrophy (DMD) is an X‐linked recessive muscle wasting disorder characterised by the absence of the protein dystrophin. Antisense oligonucleotides have been used to re‐direct dystrophin pre‐mRNA processing by blocking sequences crucial to pre‐mRNA splic