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

Authors

M Carlon
Giulia Maule (ORCID: https://orcid.org/0000-0002-7770-5688)
Daniela Guidone (ORCID: https://orcid.org/0000-0002-9301-8095)
Annalisa Santini (ORCID: https://orcid.org/0009-0004-8186-5539)
Luis J. V. Galietta (ORCID: https://orcid.org/0000-0001-8686-3461)
Irene Carrozzo (ORCID: https://orcid.org/0009-0005-8152-757X)
Mattijs Bulcaen
Matteo Ciciani (ORCID: https://orcid.org/0000-0003-2045-5435)
Alessandro Umbach (ORCID: https://orcid.org/0000-0003-3681-3110)
Anna Cereseto (ORCID: https://orcid.org/0000-0003-4453-2597)
Daniele Arosio (ORCID: https://orcid.org/0000-0003-2872-6906)
Anabela Ramalho
François Vermeulen
Enrica Brugnara (ORCID: https://orcid.org/0009-0009-9314-6976)

Institutions

University of Trento (IT)
Rega Institute for Medical Research (BE)
Institute of Biophysics (BG)
Telethon Institute Of Genetics And Medicine (IT)
KU Leuven (BE)

Publication Details

Journal: Science Translational Medicine
Published: 2026-04-22
DOI: https://doi.org/10.1126/scitranslmed.adw8886
Primary Topic: CRISPR and Genetic Engineering
Type: article
Field-Weighted Citation Impact: 0.00

Funders

Cystic Fibrosis Foundation

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article

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

M Carlon, Giulia Maule, Daniela Guidone, Annalisa Santini et al.

Apr 22, 2026

Science Translational Medicine

CRISPR and Genetic Engineering

article

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

M Carlon, Giulia Maule, Daniela Guidone, Annalisa Santini, Luis J. V. Galietta, Irene Carrozzo, Mattijs Bulcaen, Matteo Ciciani, Alessandro Umbach, Anna Cereseto, Daniele Arosio, Anabela Ramalho, François Vermeulen, Enrica Brugnara

April 22, 2026article en

Abstract

No abstract available for this paper.

Science Translational MedicineVol. 18(846)

University of Trento (IT), Rega Institute for Medical Research (BE), Institute of Biophysics (BG), Telethon Institute Of Genetics And Medicine (IT), KU Leuven (BE)

Cystic Fibrosis Foundation

Life in Land

Openalex Percentile: Top 11%

CRISPR and Genetic Engineering

DOI Source OpenAlex

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