Correction of DMD in human iPSC-derived cardiomyocytes by base-editing-induced exon skipping

Duchenne muscular dystrophy (DMD) is caused by mutations in the DMD gene. Previously, we showed that adenine base editing (ABE) can efficiently correct a nonsense point mutation in a DMD mouse model. Here, we explored the feasibility of base-editing-mediated exon skipping as a therapeutic strategy f...

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Detalles Bibliográficos
Autores principales: Wang, Peipei, Li, Haiwen, Zhu, Mandi, Han, Rena Y., Guo, Shuliang, Han, Renzhi
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/PMC9792405/
https://www.ncbi.nlm.nih.gov/pubmed/36588820
http://dx.doi.org/10.1016/j.omtm.2022.11.010

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