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

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

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

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