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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| Autori principali: | , , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
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Frontiers Media S.A.
2021-05-01
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| Serie: | Frontiers in Pharmacology |
| Soggetti: | |
| Accesso online: | https://www.frontiersin.org/articles/10.3389/fphar.2021.682299/full |
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