Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing-Reference-Cited by-同舟云学术

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Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing

Published:2023-12 Issue: Volume:31 Page:101140
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Walker Amy J.,Graham Carina,Greenwood Miriam,Woodall Maximillian,Maeshima Ruhina,O’Hara-Wright Michelle,Sanz David J.,Guerrini Ileana,Aldossary Ahmad M.,O’Callaghan Christopher,Baines Deborah L.,Harrison Patrick T.,Hart Stephen L.^ORCID

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference74 articles.

1. Cystic Fibrosis;Leitch;J. R. Coll. Physicians Edinb.,2013

2. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA;Riordan;Science,1989

3. Identification of the cystic fibrosis gene: chromosome walking and jumping;Rommens;Science,1989

4. Cystic fibrosis;Boat,1989

5. New horizons for cystic fibrosis treatment;Fajac;Pharmacol. Ther.,2017

Cited by 2 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Readthrough-induced misincorporated amino acid ratios guide mutant-specific therapeutic approaches for two CFTR nonsense mutations;Frontiers in Pharmacology;2024-04-25

2. Genetic surgery for a cystic fibrosis-causing splicing mutation;Molecular Therapy - Methods & Clinical Development;2024-03

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