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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: DMPK in DM1 and CNBP in DM2. Multiple cli...
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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MDPI
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9499601/ https://www.ncbi.nlm.nih.gov/pubmed/36142405 http://dx.doi.org/10.3390/ijms231810491 |