Cystic fibrosis(CF)is an autosomal recessive genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator(CFTR)gene,and CF-related lung disease is the main factors of mortality.Since its discovery in 1938,treat-ment for CF has been limited to symptomatic treatments,and the emergence of CFTR modulators in 2012 has substantially im-proved health and quality of life for patients.However,due to the presence of multiple CF-causing mutations,mutation-specific tar-geted therapies are not universally applicable and cannot address the multisystemic nature of the disease.In contrast,gene therapy holds promise for all patients regardless of genotype,offering the potential for complete cure,yet efficacy remains elusive.The re-view briefly introduced the development history of CF,discussed the research progress and existing problems of CF gene therapy,in order to promote the development of CF gene therapy and provide guidance for the treatment of other complex diseases.
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