Recent Progress and Challenges in the Development of Antisense Therapies for Myotonic Dystrophy Type 1

Myotonic dystrophy type 1 (DM1) is a dominant genetic disease in which the expansion of long CTG trinucleotides in the 3′ UTR of the myotonic dystrophy protein kinase (DMPK) gene results in toxic RNA gain-of-function and gene mis-splicing affecting mainly the muscles, the heart, and the brain. The C...

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Detalles Bibliográficos
Autores principales: De Serres-Bérard, Thiéry, Ait Benichou, Siham, Jauvin, Dominic, Boutjdir, Mohamed, Puymirat, Jack, Chahine, Mohamed
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9657934/
https://www.ncbi.nlm.nih.gov/pubmed/36362145
http://dx.doi.org/10.3390/ijms232113359

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9657934/
https://www.ncbi.nlm.nih.gov/pubmed/36362145
http://dx.doi.org/10.3390/ijms232113359

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