摘要：Cystic fibrosis is a hereditary disease caused by mutation in cystic fibrosis transmembrane conductance regulator (CFTR) gene, which controls CFTR proteins. Many are or have been threatened by this rare but life-threating disease, therefore, more attention should be paid to better solve this public health issue. Here we provide a thorough review about cystic fibrosis in the aspects of disease pathogenesis, clinical manifestation as well as clinical therapeutics to achieve a deeper understanding of this disease. Among the demand of innovative clinical strategies, appearance of the combination drug trikafta is a promising sign of better dealing with cystic fibrosis.
其他摘要：Cystic fibrosis is a hereditary disease caused by mutation in cystic fibrosis transmembrane conductance regulator (CFTR) gene, which controls CFTR proteins. Many are or have been threatened by this rare but life-threating disease, therefore, more attention should be paid to better solve this public health issue. Here we provide a thorough review about cystic fibrosis in the aspects of disease pathogenesis, clinical manifestation as well as clinical therapeutics to achieve a deeper understanding of this disease. Among the demand of innovative clinical strategies, appearance of the combination drug trikafta is a promising sign of better dealing with cystic fibrosis.
