Non-uniform dystrophin re-expression after CRISPR-mediated exon excision in the dystrophin/utrophin double-knockout mouse model of DMD

Duchenne muscular dystrophy (DMD) is the most prevalent inherited myopathy affecting children, caused by genetic loss of the gene encoding the dystrophin protein. Here we have investigated the use of the Staphylococcus aureus CRISPR-Cas9 system and a double-cut strategy, delivered using a pair of ad...

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Detalles Bibliográficos
Autores principales: Hanson, Britt, Stenler, Sofia, Ahlskog, Nina, Chwalenia, Katarzyna, Svrzikapa, Nenad, Coenen-Stass, Anna M.L., Weinberg, Marc S., Wood, Matthew J.A., Roberts, Thomas C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9664411/
https://www.ncbi.nlm.nih.gov/pubmed/36420212
http://dx.doi.org/10.1016/j.omtn.2022.10.010

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9664411/
https://www.ncbi.nlm.nih.gov/pubmed/36420212
http://dx.doi.org/10.1016/j.omtn.2022.10.010

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