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Functional correction of the untreatable CFTR 1717-1G>A mutation through mRNA- and sgRNA-optimized base editing.

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Title: Functional correction of the untreatable CFTR 1717-1G>A mutation through mRNA- and sgRNA-optimized base editing.
Authors: Umbach A; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy., Santini A; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy., Bulcaen M; Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, B-3000 Leuven, Belgium.; BREATHE, Department of Chronic Diseases and Metabolism, KU Leuven, B-3000 Leuven, Belgium., Guidone D; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Naples, Italy., Maule G; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy., Arosio D; Institute of Biophysics, CNR, Via alla Cascata 56/C, 38123 Trento, Italy., Carrozzo I; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy., Ciciani M; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy., Brugnara E; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy., Ramalho A; Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium., Vermeulen F; Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium., Galietta LJV; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Naples, Italy., Carlon MS; BREATHE, Department of Chronic Diseases and Metabolism, KU Leuven, B-3000 Leuven, Belgium., Cereseto A; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy.
Source: Science translational medicine [Sci Transl Med] 2026 Apr 22; Vol. 18 (846), pp. eadw8886. Date of Electronic Publication: 2026 Apr 22.
Publication Type: Journal Article
Journal Info: Publisher: American Association for the Advancement of Science Country of Publication: United States NLM ID: 101505086 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1946-6242 (Electronic) Linking ISSN: 19466234 NLM ISO Abbreviation: Sci Transl Med Subsets: MEDLINE
Database: MEDLINE Ultimate
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  Data: Functional correction of the untreatable CFTR 1717-1G>A mutation through mRNA- and sgRNA-optimized base editing.
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  Data: <searchLink fieldCode="AU" term="%22Umbach+A%22">Umbach A</searchLink>; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy.<br /><searchLink fieldCode="AU" term="%22Santini+A%22">Santini A</searchLink>; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy.<br /><searchLink fieldCode="AU" term="%22Bulcaen+M%22">Bulcaen M</searchLink>; Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, B-3000 Leuven, Belgium.; BREATHE, Department of Chronic Diseases and Metabolism, KU Leuven, B-3000 Leuven, Belgium.<br /><searchLink fieldCode="AU" term="%22Guidone+D%22">Guidone D</searchLink>; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Maule+G%22">Maule G</searchLink>; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy.<br /><searchLink fieldCode="AU" term="%22Arosio+D%22">Arosio D</searchLink>; Institute of Biophysics, CNR, Via alla Cascata 56/C, 38123 Trento, Italy.<br /><searchLink fieldCode="AU" term="%22Carrozzo+I%22">Carrozzo I</searchLink>; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy.<br /><searchLink fieldCode="AU" term="%22Ciciani+M%22">Ciciani M</searchLink>; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy.<br /><searchLink fieldCode="AU" term="%22Brugnara+E%22">Brugnara E</searchLink>; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy.<br /><searchLink fieldCode="AU" term="%22Ramalho+A%22">Ramalho A</searchLink>; Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium.<br /><searchLink fieldCode="AU" term="%22Vermeulen+F%22">Vermeulen F</searchLink>; Department of Development and Regeneration, KU Leuven, 3000 Leuven, Belgium.<br /><searchLink fieldCode="AU" term="%22Galietta+LJV%22">Galietta LJV</searchLink>; Telethon Institute of Genetics and Medicine (TIGEM), 80078 Naples, Italy.<br /><searchLink fieldCode="AU" term="%22Carlon+MS%22">Carlon MS</searchLink>; BREATHE, Department of Chronic Diseases and Metabolism, KU Leuven, B-3000 Leuven, Belgium.<br /><searchLink fieldCode="AU" term="%22Cereseto+A%22">Cereseto A</searchLink>; Department CIBIO, Laboratory of Advanced Genome Editing Technologies, University of Trento, 38123 Trento, Italy.
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  Data: <searchLink fieldCode="JN" term="%22101505086%22">Science translational medicine</searchLink> [Sci Transl Med] 2026 Apr 22; Vol. 18 (846), pp. eadw8886. <i>Date of Electronic Publication: </i>2026 Apr 22.
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