CRISPR-Cas9 homology-independent targeted integration of exons 1–19 restores full-length dystrophin in mice

Duchenne muscular dystrophy is an X-linked disorder typically caused by out-of-frame mutations in the DMD gene. Most of these are deletions of one or more exons, which can theoretically be corrected through CRISPR-Cas9-mediated knockin. Homology-independent targeted integration is a mechanism for ac...

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Detalles Bibliográficos
Autores principales: Stephenson, Anthony A., Nicolau, Stefan, Vetter, Tatyana A., Dufresne, Gabrielle P., Frair, Emma C., Sarff, Jessica E., Wheeler, Gregory L., Kelly, Benjamin J., White, Peter, Flanigan, Kevin M.
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/PMC10495553/
https://www.ncbi.nlm.nih.gov/pubmed/37706184
http://dx.doi.org/10.1016/j.omtm.2023.08.009

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