Research progress of cystic fibrosis and its therapies
10.16438/j.0513-4870.2021-1750
- VernacularTitle:囊性纤维化及其治疗方法的研究进展
- Author:
Lin LIN
1
;
Yan WANG
1
;
Chuan-bin WU
2
;
Chun-e ZHU
1
Author Information
1. School of Chinese Materia Medica, Guangdong Pharmaceutical University, Guangzhou 510006, China
2. School of Pharmaceutical Sciences, Jinan University, Guangzhou 510032, China
- Publication Type:Research Article
- Keywords:
cystic fibrosis;
cystic fibrosis transmembrane conductance regulator;
gene mutation;
CFTR modulator;
mechanism of action of drugs;
antibiotic
- From:
Acta Pharmaceutica Sinica
2022;57(7):2024-2031
- CountryChina
- Language:Chinese
-
Abstract:
Cystic fibrosis (CF) is a common and life-threatening autosomal recessive disorder in Caucasians populations. The disease is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The absence of mature CFTR at the correct cellular location or dysfunction of CFTR proteins has been observed in CF patients. CF is frequently accompanied by a variety of complex complications, such as impairments in pulmonary functions, which may lead to successive infections and respiratory failure. Recently, with the understanding of the pathogenesis of CF, a wide array of therapeutic strategies for the treatment of CF has been designed. This review summarizes pathogenic mechanisms of CF, mechanisms of action of drugs, routes of administration, and new drug development as well as provides insights into the advanced treatment strategies for CF.
