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. |
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| 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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| ISSN: | 1946-6242 |
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| DOI: | 10.1126/scitranslmed.adw8886 |
