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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書目詳細資料

Principais autores:	Isabelle Fajac, Isabelle Sermet
格式:	Artigo
語言:	Inglês
出版:	MDPI AG 2021-10-01
叢編:	Cells
主題:	cystic fibrosis CFTR modulators readthrough agents RNA therapy gene therapy gene editing
在線閱讀:	https://www.mdpi.com/2073-4409/10/10/2793
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