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Sustainable recovery of MBNL activity in autoregulatory feedback loop in myotonic dystrophy
Muscleblind-like proteins (MBNLs) are RNA-binding proteins essential for the developmental regulation of various processes including alternative splicing. Their activity is misregulated in myotonic dystrophy type 1 (DM1), an incurable genetic, neuro-muscular disorder caused by uncontrolled expansion...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2022
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9672890/ https://www.ncbi.nlm.nih.gov/pubmed/36420218 http://dx.doi.org/10.1016/j.omtn.2022.10.023 |