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
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Authors
Bibliographic Details
- Journal:
- Human gene therapy
- Volume:
- 24
- Issue:
- 5
- Pages:
- 479-488
- Publication date:
- 2013-05-01
- DOI:
- EISSN:
-
1557-7422
- ISSN:
-
1043-0342
- Source identifiers:
-
391063
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
pubs:391063
- UUID:
-
uuid:88092950-e4f6-4b22-98cb-1de316d3af9d
- Local pid:
- pubs:391063
- Deposit date:
- 2013-12-13
Terms of use
- Copyright date:
- 2013
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