Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor-Reference-Cited by-同舟云学术

Functional restoration of a CFTR splicing mutation through RNA delivery of CRISPR adenine base editor

Published:2023-06 Issue:6 Volume:31 Page:1647-1660
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Amistadi Simone,Maule Giulia^ORCID,Ciciani Matteo,Ensinck Marjolein M.,De Keersmaecker Liesbeth,Ramalho Anabela S.,Guidone Daniela,Buccirossi Martina,Galietta Luis J.V.,Carlon Marianne S.,Cereseto Anna^ORCID

Funder

Horizon 2020

Fondazione per La Ricerca sulla Fibrosi Cistica

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference69 articles.

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3. CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine;Lopes-Pacheco;Front. Pharmacol.,2019

4. Disease progression in patients with cystic fibrosis treated with ivacaftor: data from national US and UK registries;Volkova;J. Cyst. Fibros.,2020

5. Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles;Taylor-Cousar;ERJ Open Res.,2019

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1. Functional rescue of F508del-CFTR through revertant mutations introduced by CRISPR base editing;2024-08-29

2. A new era of targeting cystic fibrosis with non-viral delivery of genomic medicines;Advanced Drug Delivery Reviews;2024-06

3. The research progress of correcting pathogenic mutations by base editing;Obstetrics and Gynecology;2024-05-06

4. Prime editing functionally corrects cystic fibrosis-causing CFTR mutations in human organoids and airway epithelial cells;Cell Reports Medicine;2024-05

5. Exploring Mechanisms of Lipid Nanoparticle‐Mucus Interactions in Healthy and Cystic Fibrosis Conditions;Advanced Healthcare Materials;2024-04-13