Therapeutic Approaches for Patients with Cystic Fibrosis Not Eligible for Current CFTR Modulators

Cystic fibrosis is a severe autosomal recessive disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (<i>CFTR</i>) gene encoding the CFTR protein, a chloride channel expressed in many epithelial cells. New drugs called CFTR modulators aim at restoring the CFTR prot...

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Autors principals:	Isabelle Fajac, Isabelle Sermet
Format:	Artigo
Idioma:	Inglês
Publicat:	MDPI AG 2021-10-01
Col·lecció:	Cells
Matèries:	cystic fibrosis CFTR modulators readthrough agents RNA therapy gene therapy gene editing
Accés en línia:	https://www.mdpi.com/2073-4409/10/10/2793
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