Current landscape of cystic fibrosis gene therapy

Cystic fibrosis is a life-threatening disease that is caused by mutations in CFTR, a gene which encodes an ion channel that supports proper function of several epithelial tissues, most critically the lung. Without CFTR, airway barrier mechanisms are impaired, allowing for chronic, recurrent infectio...

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Main Authors: Lindsey W. Plasschaert (Author), Kelvin D. MacDonald (Author), Jeffrey S. Moffit (Author)
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出版: Frontiers Media S.A., 2024-10-01T00:00:00Z.
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