囊性纤维化肺病的当前及未来治疗选择：最新证据与临床意义
Current and future treatment options for cystic fibrosis lung disease: latest evidence and clinical implications.
作者信息
Edmondson Claire, Davies Jane C
机构信息
Royal Brompton & Harefield NHS Foundation Trust, Paediatric Respiratory Medicine, London, UK.
Imperial College London, Paediatric Respirology and Experimental Medicine, London SW7 2AZ, UK.
出版信息
Ther Adv Chronic Dis. 2016 May;7(3):170-83. doi: 10.1177/2040622316641352. Epub 2016 May 1.
Abstract
Treatment for cystic fibrosis (CF) has conventionally targeted downstream consequences of the defect such as mucus plugging and infection. More recently, significant advances have been made in treating the root cause of the disease, namely a defective CF transmembrane conductance regulator (CFTR) gene. This review summarizes current pulmonary treatment options and highlights advances in research and development of new therapies.
摘要
传统上，囊性纤维化（CF）的治疗针对该缺陷的下游后果，如黏液阻塞和感染。最近，在治疗该疾病的根本原因方面取得了重大进展，即囊性纤维化跨膜传导调节因子（CFTR）基因缺陷。本综述总结了当前的肺部治疗选择，并重点介绍了新疗法研发方面的进展。
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