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Combined treatment with peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) and AAV-U7 rescues the severe DMD phenotype in mice

Abstract:

Duchenne muscular dystrophy (DMD) is a devastating neuromuscular disease caused by an absence of the dystrophin protein, which is essential for muscle fiber integrity. Among the developed therapeutic strategies for DMD, exon skipping approach corrects the frame shift and partially restores dystrophin expression. It could be achieved through the use of antisense sequences, such as peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) or the small nuclear RNA-U7 carried by an adeno-a...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1016/j.omtm.2020.03.011

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Institution:
University of Oxford
Division:
MSD
Department:
Paediatrics
Role:
Author
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Publisher:
Elsevier Publisher's website
Journal:
Molecular Therapy - Methods and Clinical Development Journal website
Volume:
17
Pages:
695-708
Publication date:
2020-03-17
Acceptance date:
2020-02-24
DOI:
EISSN:
2329-0501
ISSN:
2329-0501
Language:
English
Keywords:
Pubs id:
1088970
Local pid:
pubs:1088970
Deposit date:
2020-02-25

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