囊性纤维化
医学
囊性纤维化跨膜传导调节器
遗传增强
生物信息学
重症监护医学
内科学
基因
遗传学
生物
标识
DOI:10.1016/s2213-2600(22)00253-3
摘要
There have been transformative changes in cystic fibrosis treatment in the past 10 years. The introduction of cystic fibrosis transmembrane regulator (CFTR) modulators revolutionised therapy for many people with cystic fibrosis. Despite representing a huge breakthrough, CFTR modulators are mutation specific; thus, ineffective in approximately 10% of people with rare genotypes, with some studies suggesting it could be 20–30% among Asian, Hispanic, and Black people. There is considerable interest in alternative treatment approaches that potentially promise universal effectiveness.
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