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Article

Natural Compound Boldine Lessens Myotonic Dystrophy Type 1 Phenotypes in DM1 Drosophila Models, Patient-Derived Cell Lines, and HSALR Mice.

International journal of molecular sciences - 6 Jun 2023

Álvarez-Abril Mari Carmen, García-Alcover Irma, Colonques-Bellmunt Jordi, Garijo Raquel, Pérez-Alonso Manuel, Artero Rubén, López-Castel Arturo

Abstract excerpt

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 repeat in the DMPK gene's 3' UTR, has led to exploring various therapeutic approaches in recent years, a few of which...

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PMID: 37372969
DOI: 10.3390/ijms24129820
PMCID: PMC10298378

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