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Journal article

Application of CRISPR-Cas9-mediated genome editing for the treatment of myotonic dystrophy type 1

Abstract:

Myotonic dystrophy type 1 (DM1) is a debilitating multisystemic disorder, caused by expansion of a CTG microsatellite repeat in the 3ʹ untranslated region of the DMPK (dystrophia myotonica protein kinase) gene. To date, novel therapeutic approaches have focused on transient suppression of the mutant, repeat-expanded RNA. However, recent developments in the field of genome editing have raised the exciting possibility of inducing permanent correction of the DM1 genetic defect. Specifically, rep...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1016/j.ymthe.2020.10.005

Authors


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Institution:
University of Oxford
Division:
MSD
Department:
Doctoral Training Centre - MSD
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Physiology Anatomy & Genetics
Role:
Author
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Paediatrics
Role:
Author
ORCID:
0000-0002-5436-6011
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Paediatrics
Role:
Author
ORCID:
0000-0003-4376-9381
More by this author
Institution:
University of Oxford
Division:
MSD
Department:
Paediatrics
Role:
Author
Publisher:
Cell Press
Journal:
Molecular Therapy More from this journal
Volume:
28
Issue:
12
Pages:
2527-2539
Publication date:
2020-10-14
Acceptance date:
2020-09-04
DOI:
EISSN:
1525-0024
ISSN:
1525-0016
Language:
English
Keywords:
Pubs id:
1139149
Local pid:
pubs:1139149
Deposit date:
2020-10-22

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