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Use of adenine base editing and homology-independent targeted integration strategies to correct the cystic fibrosis causing variant, W1282X.
Mention, Karen; Cavusoglu-Doran, Kader; Joynt, Anya T; Santos, Lúcia; Sanz, David; Eastman, Alice C; Merlo, Christian; Langfelder-Schwind, Elinor; Scallan, Martina F; Farinha, Carlos M; Cutting, Garry R; Sharma, Neeraj; Harrison, Patrick T.
Afiliação
  • Mention K; Department of Physiology, University College Cork, College Road, Cork, T12 K8AF, Ireland.
  • Cavusoglu-Doran K; School of Microbiology, University College Cork, College Road, Cork, T12 K8AF, Ireland.
  • Joynt AT; Department of Physiology, University College Cork, College Road, Cork, T12 K8AF, Ireland.
  • Santos L; McKusick-Nathans Department of Genetic Medicine, Johns Hopkins University, 3400 N. Charles Street, Baltimore, MD 21218, United States.
  • Sanz D; Department of Physiology, University College Cork, College Road, Cork, T12 K8AF, Ireland.
  • Eastman AC; Faculty of Sciences, BioISI - Biosystems & Integrative Sciences Institute, University of Lisboa, Campo Grande, C8 bdg, Lisboa 1749-016, Portugal.
  • Merlo C; Department of Physiology, University College Cork, College Road, Cork, T12 K8AF, Ireland.
  • Langfelder-Schwind E; McKusick-Nathans Department of Genetic Medicine, Johns Hopkins University, 3400 N. Charles Street, Baltimore, MD 21218, United States.
  • Scallan MF; Division of Pulmonary and Critical Care Medicine, Department of Medicine, Johns Hopkins Hospital, 1800 Orleans St, Baltimore, MD 21287, United States.
  • Farinha CM; The Cystic Fibrosis Center, Lenox Hill Hospital, 100 E. 77th Street, 4E, New York, NY 10075, United States.
  • Cutting GR; School of Microbiology, University College Cork, College Road, Cork, T12 K8AF, Ireland.
  • Sharma N; Faculty of Sciences, BioISI - Biosystems & Integrative Sciences Institute, University of Lisboa, Campo Grande, C8 bdg, Lisboa 1749-016, Portugal.
  • Harrison PT; McKusick-Nathans Department of Genetic Medicine, Johns Hopkins University, 3400 N. Charles Street, Baltimore, MD 21218, United States.
Hum Mol Genet ; 32(23): 3237-3248, 2023 Nov 17.
Article em En | MEDLINE | ID: mdl-37649273
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Fibrose Cística Idioma: En Ano de publicação: 2023 Tipo de documento: Article
Texto completo
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Fibrose Cística Idioma: En Ano de publicação: 2023 Tipo de documento: Article