Natural Compound Boldine Lessens Myotonic Dystrophy Type 1 Phenotypes in DM1 Drosophila Models, Patient-Derived Cell Lines, and HSA<sup>LR</sup> Mice

Natural Compound Boldine Lessens Myotonic Dystrophy Type 1 Phenotypes in DM1 Drosophila Models, Patient-Derived Cell Lines, and HSA<sup>LR</sup> Mice

Myotonic dystrophy type 1 (DM1) is a complex rare disorder characterized by progressive muscle dysfunction, involving weakness, myotonia, and wasting, but also exhibiting additional clinical signs in multiple organs and systems. Central dysregulation, caused by an expansion of a CTG trinucleotide re...

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Bibliographic Details
Main Authors: Mari Carmen Álvarez-Abril, Irma García-Alcover, Jordi Colonques-Bellmunt, Raquel Garijo, Manuel Pérez-Alonso, Rubén Artero, Arturo López-Castel
Format: Article
Language:English
Published: MDPI AG 2023-06-01
Series:International Journal of Molecular Sciences
Subjects:
boldine
myotonic dystrophy
rare disease
Drosophila
drug development
natural small molecule
Online Access:https://www.mdpi.com/1422-0067/24/12/9820

Internet

https://www.mdpi.com/1422-0067/24/12/9820

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