Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells

Myotonic dystrophy type 1 (DM1) is a neuromuscular disease that originates from an expansion of CTG microsatellites in the 3′ untranslated region of the DMPK gene, thus leading to the expression of transcripts containing expanded CUG repeats (CUGexp). The pathophysiology is explained by a toxic RNA...

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Detalles Bibliográficos
Autores principales: Porquet, Florent, Weidong, Lin, Jehasse, Kévin, Gazon, Hélène, Kondili, Maria, Blacher, Silvia, Massotte, Laurent, Di Valentin, Emmannuel, Furling, Denis, Gillet, Nicolas Albert, Klein, Arnaud François, Seutin, Vincent, Willems, Luc
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10238591/
https://www.ncbi.nlm.nih.gov/pubmed/37273786
http://dx.doi.org/10.1016/j.omtn.2023.05.007

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10238591/
https://www.ncbi.nlm.nih.gov/pubmed/37273786
http://dx.doi.org/10.1016/j.omtn.2023.05.007

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