Journal article
A fusion peptide directs enhanced systemic dystrophin exon skipping and functional restoration in dystrophin-deficient mdx mice.
- Abstract:
-
Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene that abolish the synthesis of dystrophin protein. Antisense oligonucleotides (AOs) targeted to trigger excision of an exon bearing a mutant premature stop codon in the DMD transcript have been shown to skip the mutated exon and partially restore functional dystrophin protein in dystrophin-deficient mdx mice. To fully exploit the therapeutic potential of this method requires highly efficient systemic AO delivery to multip...
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- Publisher copy:
- 10.1093/hmg/ddp395
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Authors
Bibliographic Details
- Journal:
- Human molecular genetics
- Volume:
- 18
- Issue:
- 22
- Pages:
- 4405-4414
- Publication date:
- 2009-11-01
- DOI:
- EISSN:
-
1460-2083
- ISSN:
-
0964-6906
- Source identifiers:
-
113439
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
pubs:113439
- UUID:
-
uuid:ce3ccce1-dad8-4f30-a9b5-f4938002f1af
- Local pid:
- pubs:113439
- Deposit date:
- 2012-12-19
Terms of use
- Copyright date:
- 2009
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