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With newer tools for gene editing, is it time to revisit genetic therapy for cystic fibrosis?

With newer tools for gene editing, is it time to revisit genetic therapy for cystic fibrosis?

Anuj Gupta
Ankita Gupta
Larissa Boyd
Genes & Diseases第8卷, 第2期pp.168-170纸质出版 2021-03-01在线发表 2020-01-22
120900

Cystic Fibrosis (CF) is an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene on chromosome 7. These abnormalities affect the chloride channels in the mucus producing epithelial cells. With these channels not working properly, water is unable to leave the cells to enter the mucus; thus, leading to sticky mucus which obstructs airways and ducts in the body.