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Journal article

PRO-051, an antisense oligonucleotide for the potential treatment of Duchenne muscular dystrophy.

Abstract:

PRO-051 (GSK-2402968), being developed by GlaxoSmithKline plc, under license from Leiden University Medical Center and Prosensa Therapeutics BV, is a 2'-O-methyl phosphorothioate antisense oligonucleotide for the potential treatment of Duchenne muscular dystrophy (DMD). The PRO-051 oligonucleotide sequence induces skipping of exon 51 of the dystrophin gene by binding to a sequence within the dystrophin pre-mRNA and masking the exon inclusion signals that are used for splicing. Removal of exon...

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Publication status:
Published

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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Role:
Author
More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Role:
Author
Journal:
Current opinion in molecular therapeutics
Volume:
12
Issue:
4
Pages:
478-486
Publication date:
2010-08-05
EISSN:
2040-3445
ISSN:
1464-8431
URN:
uuid:8d7779cd-1a4b-4274-857d-72b3d45f02a0
Source identifiers:
113552
Local pid:
pubs:113552

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