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Prevention of dystrophic pathology in severely affected dystrophin/utrophin-deficient mice by morpholino-oligomer-mediated exon-skipping.

Abstract:

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder caused by mutations in the dystrophin gene that result in the absence of functional protein. Antisense-mediated exon-skipping is one of the most promising approaches for the treatment of DMD because of its capacity to correct the reading frame and restore dystrophin expression, which has been demonstrated in vitro and in vivo. In particular, peptide-conjugated phosphorodiamidate morpholino oligomers (PPMOs) have recently bee...

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Publication status:
Published

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Publisher copy:
10.1038/mt.2009.248

Authors


Goyenvalle, A More by this author
Fletcher, S More by this author
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Journal:
Molecular therapy : the journal of the American Society of Gene Therapy
Volume:
18
Issue:
1
Pages:
198-205
Publication date:
2010-01-05
DOI:
EISSN:
1525-0024
ISSN:
1525-0016
URN:
uuid:a2bb929c-7bec-4f90-a644-e05f09c3b20a
Source identifiers:
106392
Local pid:
pubs:106392

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