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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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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
Language:
English
Keywords:
Pubs id:
pubs:113439
UUID:
uuid:ce3ccce1-dad8-4f30-a9b5-f4938002f1af
Local pid:
pubs:113439
Deposit date:
2012-12-19

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