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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
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Journal:
Current pharmaceutical design
Volume:
19
Issue:
16
Pages:
2948-2962
Publication date:
2013
DOI:
EISSN:
1873-4286
ISSN:
1381-6128
URN:
uuid:399113dd-8bba-49d3-902a-cdbe1508c2b9
Source identifiers:
363844
Local pid:
pubs:363844

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