Prime editing-mediated correction of the CFTR W1282X mutation in iPSCs and derived airway epithelial cells

A major unmet need in the cystic fibrosis (CF) therapeutic landscape is the lack of effective treatments for nonsense CFTR mutations, which affect approximately 10% of CF patients. Correction of nonsense CFTR mutations via genomic editing represents a promising therapeutic approach. In this study, w...

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Detalles Bibliográficos
Autores principales: Li, Chao, Liu, Zhong, Anderson, Justin, Liu, Zhongyu, Tang, Liping, Li, Yao, Peng, Ning, Chen, Jianguo, Liu, Xueming, Fu, Lianwu, Townes, Tim M., Rowe, Steven M., Bedwell, David M., Guimbellot, Jennifer, Zhao, Rui
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2023
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10686454/
https://www.ncbi.nlm.nih.gov/pubmed/38019847
http://dx.doi.org/10.1371/journal.pone.0295009

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