Time-controlled and muscle-specific CRISPR/Cas9-mediated deletion of CTG-repeat expansion in the DMPK gene

CRISPR/Cas9-mediated therapeutic gene editing is a promising technology for durable treatment of incurable monogenic diseases such as myotonic dystrophies. Gene-editing approaches have been recently applied to in vitro and in vivo models of myotonic dystrophy type 1 (DM1) to delete the pathogenic CT...

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Detalles Bibliográficos
Autores principales: Cardinali, Beatrice, Provenzano, Claudia, Izzo, Mariapaola, Voellenkle, Christine, Battistini, Jonathan, Strimpakos, Georgios, Golini, Elisabetta, Mandillo, Silvia, Scavizzi, Ferdinando, Raspa, Marcello, Perfetti, Alessandra, Baci, Denisa, Lazarevic, Dejan, Garcia-Manteiga, Jose Manuel, Gourdon, Geneviève, Martelli, Fabio, Falcone, Germana
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8693309/
https://www.ncbi.nlm.nih.gov/pubmed/34976437
http://dx.doi.org/10.1016/j.omtn.2021.11.024

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8693309/
https://www.ncbi.nlm.nih.gov/pubmed/34976437
http://dx.doi.org/10.1016/j.omtn.2021.11.024

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