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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Autores principales:	Giulia Maule, Antonio Casini, Claudia Montagna, Anabela S. Ramalho, Kris De Boeck, Zeger Debyser, Marianne S. Carlon, Gianluca Petris, Anna Cereseto
Formato:	article
Lenguaje:	EN
Publicado:	Nature Portfolio 2019
Materias:	Science Q
Acceso en línea:	https://doaj.org/article/d7c2cba4da6b42e3a01044d6646c73ee
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spelling	oai:doaj.org-article:d7c2cba4da6b42e3a01044d6646c73ee2021-12-02T15:35:47ZAllele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing10.1038/s41467-019-11454-92041-1723https://doaj.org/article/d7c2cba4da6b42e3a01044d6646c73ee2019-08-01T00:00:00Zhttps://doi.org/10.1038/s41467-019-11454-9https://doaj.org/toc/2041-1723Cystic 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 and airway epithelial cells.Giulia MauleAntonio CasiniClaudia MontagnaAnabela S. RamalhoKris De BoeckZeger DebyserMarianne S. CarlonGianluca PetrisAnna CeresetoNature PortfolioarticleScienceQENNature Communications, Vol 10, Iss 1, Pp 1-11 (2019)
institution	DOAJ
collection	DOAJ
language	EN
topic	Science Q
spellingShingle	Science Q Giulia Maule Antonio Casini Claudia Montagna Anabela S. Ramalho Kris De Boeck Zeger Debyser Marianne S. Carlon Gianluca Petris Anna Cereseto Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
description	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 and airway epithelial cells.
format	article
author	Giulia Maule Antonio Casini Claudia Montagna Anabela S. Ramalho Kris De Boeck Zeger Debyser Marianne S. Carlon Gianluca Petris Anna Cereseto
author_facet	Giulia Maule Antonio Casini Claudia Montagna Anabela S. Ramalho Kris De Boeck Zeger Debyser Marianne S. Carlon Gianluca Petris Anna Cereseto
author_sort	Giulia Maule
title	Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
title_short	Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
title_full	Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
title_fullStr	Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
title_full_unstemmed	Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing
title_sort	allele specific repair of splicing mutations in cystic fibrosis through ascas12a genome editing
publisher	Nature Portfolio
publishDate	2019
url	https://doaj.org/article/d7c2cba4da6b42e3a01044d6646c73ee
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Allele specific repair of splicing mutations in cystic fibrosis through AsCas12a genome editing

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