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

Cystic fibrosis (CF) is an autosomal recessive disease caused by mutations in the CFTR gene. The 3272–26A>G and 3849+10kbC>T CFTR mutations alter the correct splicing of the CFTR gene, generating new acceptor and donor splice sites respectively. Here we develop a genome editing approach to per...

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Detalles Bibliográficos
Autores principales: Maule, Giulia, Casini, Antonio, Montagna, Claudia, Ramalho, Anabela S., De Boeck, Kris, Debyser, Zeger, Carlon, Marianne S., Petris, Gianluca, Cereseto, Anna
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6685978/
https://www.ncbi.nlm.nih.gov/pubmed/31391465
http://dx.doi.org/10.1038/s41467-019-11454-9

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6685978/
https://www.ncbi.nlm.nih.gov/pubmed/31391465
http://dx.doi.org/10.1038/s41467-019-11454-9

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