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

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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
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  Data: Molecular and functional correction of a deep intronic splicing mutation in CFTR by CRISPR-Cas9 gene editing.
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  Data: <searchLink fieldCode="AU" term="%22Walker+AJ%22">Walker AJ</searchLink>; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Graham+C%22">Graham C</searchLink>; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Greenwood+M%22">Greenwood M</searchLink>; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Woodall+M%22">Woodall M</searchLink>; Institute for Infection and Immunity, St. George's, University of London, London, UK.<br /><searchLink fieldCode="AU" term="%22Maeshima+R%22">Maeshima R</searchLink>; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22O'Hara-Wright+M%22">O'Hara-Wright M</searchLink>; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Sanz+DJ%22">Sanz DJ</searchLink>; Department of Physiology, BioSciences Institute, University College Cork, Cork, Ireland.<br /><searchLink fieldCode="AU" term="%22Guerrini+I%22">Guerrini I</searchLink>; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Aldossary+AM%22">Aldossary AM</searchLink>; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22O'Callaghan+C%22">O'Callaghan C</searchLink>; Infection, Immunity & Inflammation Department, UCL Great Ormond Street Institute of Child Health, London, UK.<br /><searchLink fieldCode="AU" term="%22Baines+DL%22">Baines DL</searchLink>; Institute for Infection and Immunity, St. George's, University of London, London, UK.<br /><searchLink fieldCode="AU" term="%22Harrison+PT%22">Harrison PT</searchLink>; Department of Physiology, BioSciences Institute, University College Cork, Cork, Ireland.<br /><searchLink fieldCode="AU" term="%22Hart+SL%22">Hart SL</searchLink>; Genetics and Genomic Medicine Department, UCL Great Ormond Street Institute of Child Health, London, UK.
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  Data: <searchLink fieldCode="JN" term="%22101624857%22">Molecular therapy. Methods & clinical development</searchLink> [Mol Ther Methods Clin Dev] 2023 Oct 18; Vol. 31, pp. 101140. <i>Date of Electronic Publication: </i>2023 Oct 18 (<i>Print Publication: </i>2023).
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  Data: <i>Publisher: </i><searchLink fieldCode="PB" term="%22Cell+Press%22">Cell Press </searchLink><i>Country of Publication: </i>United States <i>NLM ID: </i>101624857 <i>Publication Model: </i>eCollection <i>Cited Medium: </i>Print <i>ISSN: </i>2329-0501 (Print) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2223290501%22">23290501 </searchLink><i>NLM ISO Abbreviation: </i>Mol Ther Methods Clin Dev <i>Subsets: </i>PubMed not MEDLINE
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