Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing

Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing

Cystic fibrosis is caused by mutations in the CFTR chloride channel. Here, the authors develop a gene therapy approach using the programmable nuclease AsCas12a to correct a splicing mutation in CFTR, and show efficient repair of the mutation and recovery of CFTR function in patient-derived organoids...

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Bibliographic Details
Main Authors: Giulia Maule, Antonio Casini, Claudia Montagna, Anabela S. Ramalho, Kris De Boeck, Zeger Debyser, Marianne S. Carlon, Gianluca Petris, Anna Cereseto
Format: Article
Language:English
Published: Nature Portfolio 2019-08-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-019-11454-9

Internet

https://doi.org/10.1038/s41467-019-11454-9

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