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Gene Therapy: A Possible Alternative to CFTR Modulators?

Cystic fibrosis (CF) is a rare genetic disease that affects several organs, but lung disease is the major cause of morbidity and mortality. The gene responsible for CF, the CFTR (Cystic Fibrosis Transmembrane Conductance Regulator) gene, has been discovered in 1989. Since then, gene therapy i.e., de...

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Detalles Bibliográficos
Autores principales: Mercier, J., Ruffin, M., Corvol, H., Guillot, L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8097140/
https://www.ncbi.nlm.nih.gov/pubmed/33967785
http://dx.doi.org/10.3389/fphar.2021.648203