Gene Therapy for Cystic Fibrosis: Lessons Learned and Paths Forward

Overview

Journal Mol Ther

Publisher Cell Press

Specialties Molecular Biology
Pharmacology

Date 2021 Jan 20

PMID 33472034

Citations 5

Authors

Soon H Choi

John F Engelhardt

Affiliations

Soon will be listed here.

Citing Articles

Artificial intelligence-guided design of lipid nanoparticles for pulmonary gene therapy.

Witten J, Raji I, Manan R, Beyer E, Bartlett S, Tang Y Nat Biotechnol. 2024; .

PMID: 39658727 DOI: 10.1038/s41587-024-02490-y.

Evaluation of anti-vector immune responses to adenovirus-mediated lung gene therapy and modulation by αCD20.

Clark R, Rabito F, Munyonho F, Remcho T, Kolls J Mol Ther Methods Clin Dev. 2024; 32(3):101286.

PMID: 39070292 PMC: 11283059. DOI: 10.1016/j.omtm.2024.101286.

Protospacer modification improves base editing of a canonical splice site variant and recovery of CFTR function in human airway epithelial cells.

Joynt A, Kavanagh E, Newby G, Mitchell S, Eastman A, Paul K Mol Ther Nucleic Acids. 2023; 33:335-350.

PMID: 37547293 PMC: 10400809. DOI: 10.1016/j.omtn.2023.06.020.

Immunosuppression reduces rAAV2.5T neutralizing antibodies that limit efficacy following repeat dosing to ferret lungs.

Tang Y, Fakhari S, Huntemann E, Feng Z, Wu P, Feng W Mol Ther Methods Clin Dev. 2023; 29:70-80.

PMID: 36950451 PMC: 10025970. DOI: 10.1016/j.omtm.2023.02.015.

Gene therapy for cystic fibrosis: Challenges and prospects.

Sui H, Xu X, Su Y, Gong Z, Yao M, Liu X Front Pharmacol. 2022; 13:1015926.

PMID: 36304167 PMC: 9592762. DOI: 10.3389/fphar.2022.1015926.

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