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Journal article

Clinical trials using antisense oligonucleotides in duchenne muscular dystrophy.

Abstract:

Duchenne muscular dystrophy (DMD) is a severe muscle wasting disorder caused by mutations in the DMD gene, affecting 1 in 3500 newborn males. Complete loss of muscle dystrophin protein causes progressive muscle weakness and heart and respiratory failure, leading to premature death. Antisense oligonucleotides (AONs) that bind to complementary sequences of the dystrophin pre-mRNA to induce skipping of the targeted exon by modulating pre-mRNA splicing are promising therapeutic agents for DMD. Su...

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Publication status:
Published

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Publisher copy:
10.1089/hum.2012.234

Authors


More by this author
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:
Human gene therapy
Volume:
24
Issue:
5
Pages:
479-488
Publication date:
2013-05-05
DOI:
EISSN:
1557-7422
ISSN:
1043-0342
URN:
uuid:88092950-e4f6-4b22-98cb-1de316d3af9d
Source identifiers:
391063
Local pid:
pubs:391063

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