Transiently expressed CRISPR/Cas9 induces wild-type dystrophin in vitro in DMD patient myoblasts carrying duplications

Among the mutations arising in the DMD gene and causing Duchenne Muscular Dystrophy (DMD), 10–15% are multi-exon duplications. There are no current therapeutic approaches with the ability to excise large multi-exon duplications, leaving this patient cohort without mutation-specific treatment. Using...

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Detalles Bibliográficos
Autores principales: Pini, Veronica, Mariot, Virginie, Dumonceaux, Julie, Counsell, John, O’Neill, Helen C., Farmer, Sarah, Conti, Francesco, Muntoni, Francesco
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8904532/
https://www.ncbi.nlm.nih.gov/pubmed/35260651
http://dx.doi.org/10.1038/s41598-022-07671-w

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