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Therapeutic Targeting of the GSK3β-CUGBP1 Pathway in Myotonic Dystrophy

Myotonic Dystrophy type 1 (DM1) is a neuromuscular disease associated with toxic RNA containing expanded CUG repeats. The developing therapeutic approaches to DM1 target mutant RNA or correct early toxic events downstream of the mutant RNA. We have previously described the benefits of the correction...

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Detalles Bibliográficos
Autores principales: Lutz, Maggie, Levanti, Miranda, Karns, Rebekah, Gourdon, Genevieve, Lindquist, Diana, Timchenko, Nikolai A., Timchenko, Lubov
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10342152/
https://www.ncbi.nlm.nih.gov/pubmed/37445828
http://dx.doi.org/10.3390/ijms241310650