Development of Therapeutic Approaches for Myotonic Dystrophies Type 1 and Type 2

Development of Therapeutic Approaches for Myotonic Dystrophies Type 1 and Type 2

Myotonic Dystrophies type 1 (DM1) and type 2 (DM2) are complex multisystem diseases without disease-based therapies. These disorders are caused by the expansions of unstable CTG (DM1) and CCTG (DM2) repeats outside of the coding regions of the disease genes: <i>DMPK</i> in DM1 and <i&...

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Bibliographic Details
Main Author: Lubov Timchenko
Format: Article
Language:English
Published: MDPI AG 2022-09-01
Series:International Journal of Molecular Sciences
Subjects:
myotonic dystrophy
congenital myotonic dystrophy
myotonic dystrophy type 2
clinical trials
Online Access:https://www.mdpi.com/1422-0067/23/18/10491

Internet

https://www.mdpi.com/1422-0067/23/18/10491

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