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Toward an oligonucleotide therapy for Duchenne muscular dystrophy: a complex development challenge.

Abstract:

Antisense oligonucleotide (AO)-mediated exon skipping is a promising new therapy for Duchenne muscular dystrophy (DMD), recently demonstrating proof of principle for restoring the absent dystrophin protein in DMD patients. However, the range of AO chemistries available for exon skipping is limited; effective systemic dystrophin protein restoration has yet to be demonstrated and will be required for disease modification in patients; and the current approach is mutation-specific, necessitating ...

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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Journal:
Science translational medicine
Volume:
2
Issue:
25
Pages:
25ps15
Publication date:
2010-03-05
DOI:
EISSN:
1946-6242
ISSN:
1946-6234
URN:
uuid:1144d6b0-884f-4f2d-a078-3042a1fa31c3
Source identifiers:
113700
Local pid:
pubs:113700

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