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Cell penetrating peptide delivery of splice directing oligonucleotides as a treatment for Duchenne muscular dystrophy.

Abstract:

Duchenne muscular dystrophy is a severe, X-linked muscle wasting disorder caused by the absence of an integral structural protein called dystrophin. This is caused by mutations or deletions in the dystrophin gene which disrupt the reading frame, thereby halting the production of a functional protein. A number of potential therapies have been investigated for the treatment of this disease including utrophin upregulation, 'stop-codon read through' aminoglycosides and adeno-associated virus gene...

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Publisher copy:
10.2174/1381612811319160009

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Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
Journal:
Current pharmaceutical design
Volume:
19
Issue:
16
Pages:
2948-2962
Publication date:
2013-01-01
DOI:
EISSN:
1873-4286
ISSN:
1381-6128
Source identifiers:
363844
Language:
English
Keywords:
Pubs id:
pubs:363844
UUID:
uuid:399113dd-8bba-49d3-902a-cdbe1508c2b9
Local pid:
pubs:363844
Deposit date:
2013-11-16

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