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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Principais autores:	Nicole Reyne, Patricia Cmielewski, Alexandra McCarron, Juliette Delhove, David Parsons, Martin Donnelley
Formato:	Artigo
Idioma:	Inglês
Publicado em:	Frontiers Media S.A. 2021-05-01
coleção:	Frontiers in Pharmacology
Assuntos:	cystic fibrosis lentival vector nasal potential difference (NPD) Rat gene therapy
Acesso em linha:	https://www.frontiersin.org/articles/10.3389/fphar.2021.682299/full
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