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Journal article

Peptide-conjugated oligonucleotides evoke long-lasting myotonic dystrophy correction in patient-derived cells and mice

Abstract:

Antisense oligonucleotides (ASOs) targeting pathologic RNAs have shown promising therapeutic corrections for many genetic diseases including myotonic dystrophy (DM1). Thus, ASO strategies for DM1 can abolish the toxic RNA gain-of-function mechanism caused by nuclear-retained mutant transcripts containing CUG expansions (CUGexp). However, systemic use of ASOs for this muscular disease remains challenging due to poor drug distribution to skeletal muscle. To overcome this limitation, we test an ...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1172/JCI128205

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Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
Publisher:
American Society for Clinical Investigation
Journal:
Journal of Clinical Investigation More from this journal
Volume:
129
Issue:
11
Pages:
4739-4744
Publication date:
2019-09-03
Acceptance date:
2019-07-09
DOI:
EISSN:
1558-8238
ISSN:
0021-9738
Pmid:
31479430
Language:
English
Keywords:
Pubs id:
pubs:1049821
UUID:
uuid:f4496df8-8ac9-4419-adf6-313465ce122c
Local pid:
pubs:1049821
Source identifiers:
1049821
Deposit date:
2019-09-17

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