Gene therapy for cystic fibrosis: Challenges and prospects
Cystic fibrosis (CF) is a life-threatening autosomal-recessive disease caused by mutations in a single gene encoding cystic fibrosis transmembrane conductance regulator (CFTR). CF effects multiple organs, and lung disease is the primary cause of mortality. The median age at death from CF is in the e...
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Frontiers Media S.A.,
2022-10-01T00:00:00Z.
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