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Functional correction of CFTR mutations in human airway epithelial cells using adenine base editors

Mutations in the CFTR gene that lead to premature stop codons or splicing defects cause cystic fibrosis (CF) and are not amenable to treatment by small-molecule modulators. Here, we investigate the use of adenine base editor (ABE) ribonucleoproteins (RNPs) that convert A•T to G•C base pairs as a the...

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Detalles Bibliográficos
Autores principales:	Krishnamurthy, Sateesh, Traore, Soumba, Cooney, Ashley L, Brommel, Christian M, Kulhankova, Katarina, Sinn, Patrick L, Newby, Gregory A, Liu, David R, McCray, Paul B
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2021
Materias:	Molecular Biology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8501978/ https://www.ncbi.nlm.nih.gov/pubmed/34520545 http://dx.doi.org/10.1093/nar/gkab788

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