Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing

Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy affecting many different body tissues, predominantly skeletal and cardiac muscles and the central nervous system. The expansion of CTG repeats in the DM1 protein-kinase (DMPK) gene is the genetic cause of the disease. The pathogen...

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Detalles Bibliográficos
Autores principales: Izzo, Mariapaola, Battistini, Jonathan, Provenzano, Claudia, Martelli, Fabio, Cardinali, Beatrice, Falcone, Germana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9101876/
https://www.ncbi.nlm.nih.gov/pubmed/35563013
http://dx.doi.org/10.3390/ijms23094622

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9101876/
https://www.ncbi.nlm.nih.gov/pubmed/35563013
http://dx.doi.org/10.3390/ijms23094622

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