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Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment.

Abstract:

Antisense oligonucleotides (AOs) are currently the most promising therapeutic intervention for Duchenne muscular dystrophy (DMD). AOs modulate dystrophin pre-mRNA splicing, thereby specifically restoring the dystrophin reading frame and generating a truncated but semifunctional dystrophin protein. Challenges in the development of this approach are the relatively poor systemic AO delivery and inefficient dystrophin correction in affected non-skeletal muscle tissues, including the heart. We hav...

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Publication status:
Published

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Publisher copy:
10.1038/mtna.2012.30

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Institution:
University of Oxford
Department:
Oxford, MSD, Physiology Anatomy and Genetics
Role:
Author
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Journal:
Molecular therapy. Nucleic acids
Volume:
1
Issue:
8
Pages:
e38
Publication date:
2012-01-01
DOI:
EISSN:
2162-2531
ISSN:
2162-2531
URN:
uuid:cd7c724a-b336-42e5-b26f-335142ad6946
Source identifiers:
364427
Local pid:
pubs:364427

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