Precise correction of Duchenne muscular dystrophy exon deletion mutations by base and prime editing

Duchenne muscular dystrophy (DMD) is a fatal muscle disease caused by the lack of dystrophin, which maintains muscle membrane integrity. We used an adenine base editor (ABE) to modify splice donor sites of the dystrophin gene, causing skipping of a common DMD deletion mutation of exon 51 (∆Ex51) in...

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Detalles Bibliográficos
Autores principales: Chemello, F., Chai, A. C., Li, H., Rodriguez-Caycedo, C., Sanchez-Ortiz, E., Atmanli, A., Mireault, A. A., Liu, N., Bassel-Duby, R., Olson, E. N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2021
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8087404/
https://www.ncbi.nlm.nih.gov/pubmed/33931459
http://dx.doi.org/10.1126/sciadv.abg4910

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