Prime editing strategies to mediate exon skipping in DMD gene

Duchenne muscular dystrophy is a rare and lethal hereditary disease responsible for progressive muscle wasting due to mutations in the DMD gene. We used the CRISPR-Cas9 Prime editing technology to develop different strategies to correct frameshift mutations in DMD gene carrying the deletion of exon...

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Detalles Bibliográficos
Autores principales: Happi Mbakam, Cedric, Roustant, Jeanne, Rousseau, Joel, Yameogo, Pouire, Lu, Yaoyao, Bigot, Anne, Mamchaoui, Kamel, Mouly, Vincent, Lamothe, Gabriel, Tremblay, Jacques P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2023
Materias:
Medicine
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10248452/
https://www.ncbi.nlm.nih.gov/pubmed/37305116
http://dx.doi.org/10.3389/fmed.2023.1128557

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10248452/
https://www.ncbi.nlm.nih.gov/pubmed/37305116
http://dx.doi.org/10.3389/fmed.2023.1128557

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