A cystic fibrosis gene editing approach that is on target
Author:
Funder
National Institutes of Health
National Heart Lung and Blood Institute Division of Intramural Research
Publisher
Elsevier BV
Reference7 articles.
1. Gene, RNA, and ASO-based therapeutic approaches in Cystic Fibrosis;Allaire;J. Cyst. Fibros.,2023
2. Investigating adverse genomic and regulatory changes caused by replacement of the full-length CFTR cDNA using Cas9 and AAV;Vaidyanathan;Mol. Ther. Nucleic Acids,2024
3. Targeted replacement of full-length CFTR in human airway stem cells by CRISPR-Cas9 for pan-mutation correction in the endogenous locus;Vaidyanathan;Mol. Ther.,2022
4. Readthrough compounds for nonsense mutations: bridging the translational gap;Spelier;Trends Mol. Med.,2023
5. High-Efficiency, Selection-free Gene Repair in Airway Stem Cells from Cystic Fibrosis Patients Rescues CFTR Function in Differentiated Epithelia;Vaidyanathan;Cell Stem Cell,2020
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