Single-Dose Lentiviral Mediated Gene Therapy Recovers CFTR Function in Cystic Fibrosis Knockout Rats

Cystic fibrosis (CF) is a genetic disease caused by mutations in the CF transmembrane conductance regulator (CFTR) gene, resulting in defective ion transport in the airways. Addition of a functioning CFTR gene into affected airway cells has the potential to be an effective treatment for lung disease...

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Bibliographic Details
Published in:Front Pharmacol
Main Authors: Reyne, Nicole, Cmielewski, Patricia, McCarron, Alexandra, Delhove, Juliette, Parsons, David, Donnelley, Martin
Format: Artigo
Language:Inglês
Published: Frontiers Media S.A. 2021
Subjects:
Pharmacology
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC8167067/
https://ncbi.nlm.nih.gov/pubmed/34084147
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3389/fphar.2021.682299
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