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Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor.
Amistadi, Simone; Maule, Giulia; Ciciani, Matteo; Ensinck, Marjolein M; De Keersmaecker, Liesbeth; Ramalho, Anabela S; Guidone, Daniela; Buccirossi, Martina; Galietta, Luis J V; Carlon, Marianne S; Cereseto, Anna.
Afiliação
  • Amistadi S; University of Trento, Department of Computational, Cellular and Integrative Biology, Laboratory of Molecular Virology, 38123 Trento, Italy.
  • Maule G; University of Trento, Department of Computational, Cellular and Integrative Biology, Laboratory of Molecular Virology, 38123 Trento, Italy. Electronic address: giulia.maule@unitn.it.
  • Ciciani M; University of Trento, Department of Computational, Cellular and Integrative Biology, Laboratory of Molecular Virology, 38123 Trento, Italy.
  • Ensinck MM; KU Leuven, Department of Pharmaceutical and Pharmacological Sciences, Laboratory for Molecular Virology and Gene Therapy, 3000 Leuven, Belgium.
  • De Keersmaecker L; KU Leuven, Department of Pharmaceutical and Pharmacological Sciences, Laboratory for Molecular Virology and Gene Therapy, 3000 Leuven, Belgium.
  • Ramalho AS; CF Research Lab, Woman and Child Unit, Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium.
  • Guidone D; Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Italy.
  • Buccirossi M; Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Italy.
  • Galietta LJV; Telethon Institute of Genetics and Medicine, 80078 Pozzuoli, Italy; Department of Translational Medical Sciences, University of Napoli "Federico II," 80138 Napoli, Italy.
  • Carlon MS; KU Leuven, Department of Pharmaceutical and Pharmacological Sciences, Laboratory for Molecular Virology and Gene Therapy, 3000 Leuven, Belgium; KU Leuven, Department of Chronic Diseases and Metabolism, BREATHE Laboratory, 3000 Leuven, Belgium.
  • Cereseto A; University of Trento, Department of Computational, Cellular and Integrative Biology, Laboratory of Molecular Virology, 38123 Trento, Italy. Electronic address: anna.cereseto@unitn.it.
Mol Ther ; 31(6): 1647-1660, 2023 06 07.
Article em En | MEDLINE | ID: mdl-36895161
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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Regulador de Condutância Transmembrana em Fibrose Cística / Fibrose Cística Idioma: En Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Itália
Texto completo
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XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Regulador de Condutância Transmembrana em Fibrose Cística / Fibrose Cística Idioma: En Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Itália