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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Authors
Bibliographic Details
- Publisher:
- Cell Press Publisher's website
- Journal:
- Molecular Therapy Journal website
- Volume:
- 28
- Issue:
- 12
- Pages:
- 2527-2539
- Publication date:
- 2020-10-14
- Acceptance date:
- 2020-09-04
- DOI:
- EISSN:
-
1525-0024
- ISSN:
-
1525-0016
Item Description
- Language:
- English
- Keywords:
- Pubs id:
-
1139149
- Local pid:
- pubs:1139149
- Deposit date:
- 2020-10-22
Terms of use
- Copyright holder:
- American Society of Gene and Cell Therapy
- Copyright date:
- 2020
- Rights statement:
- © 2020 The American Society of Gene and Cell Therapy.
- Notes:
- This is the accepted manuscript version of the article. The final version is available online from Cell Press at: https://doi.org/10.1016/j.ymthe.2020.10.005
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