Article
PKC activators improve the function of biogenesis-deficient CFTR channels.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jul 2026
Ye Shi-Wei, Liu Xi-Juan, Kang Xin-Lei, Shen Li, Cai Shi-Qing
Abstract excerpt
Cystic fibrosis (CF) is a lethal genetic disease caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR). The most prevalent disease-causing mutation, F508del, results in the production of misfolded CFTR proteins, which are prematurely degraded. Given the complex pathology of CF, a multimodal therapeutic strategy is essential. This underscores the urgent need to...
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