Journal article icon

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...

Expand abstract
Publication status:
Published

Actions


Authors


More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
Journal:
Current opinion in molecular therapeutics
Volume:
12
Issue:
4
Pages:
478-486
Publication date:
2010-08-01
EISSN:
2040-3445
ISSN:
1464-8431
Source identifiers:
113552
Language:
English
Keywords:
Pubs id:
pubs:113552
UUID:
uuid:8d7779cd-1a4b-4274-857d-72b3d45f02a0
Local pid:
pubs:113552
Deposit date:
2012-12-19

Terms of use


Views and Downloads






If you are the owner of this record, you can report an update to it here: Report update to this record

TO TOP