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

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Bibliographic Details
Title: Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing.
Authors: Walker AJ; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK., Graham C; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK., Greenwood M; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK., Woodall M; Institute for Infection and Immunity, St. George's, University of London, London, UK., Maeshima R; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK., O'Hara-Wright M; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK., Sanz DJ; Department of Physiology, BioSciences Institute, University College Cork, Cork, Ireland., Guerrini I; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK., Aldossary AM; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK., O'Callaghan C; Infection, Immunity & Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK., Baines DL; Institute for Infection and Immunity, St. George's, University of London, London, UK., Harrison PT; Department of Physiology, BioSciences Institute, University College Cork, Cork, Ireland., Hart SL; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.
Source: Molecular therapy. Methods & clinical development [Mol Ther Methods Clin Dev] 2023 Oct 18; Vol. 31, pp. 101140. Date of Electronic Publication: 2023 Oct 18 (Print Publication: 2023).
Publication Type: Journal Article
Journal Info: Publisher: Cell Press Country of Publication: United States NLM ID: 101624857 Publication Model: eCollection Cited Medium: Print ISSN: 2329-0501 (Print) Linking ISSN: 23290501 NLM ISO Abbreviation: Mol Ther Methods Clin Dev Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2329-0501
DOI:10.1016/j.omtm.2023.101140