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- W4308175635 abstract "Cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy brings hope to most patients with cystic fibrosis (CF), but not all. For approximately 12% of CF patients with premature termination codon mutations, large deletions, insertions, and frameshifts, the CFTR modulator therapy is not effective. Many believe that genetic-based therapies such as RNA therapies, DNA therapies, and gene editing technologies will be needed to treat mutations that are not responsive to modulator therapy. Delivery of these therapeutic agents to affected cells is the major challenge that will need to be overcome if we are to harness the power of these emerging therapies for the treatment of CF." @default.
- W4308175635 created "2022-11-08" @default.
- W4308175635 creator A5085335419 @default.
- W4308175635 date "2022-12-01" @default.
- W4308175635 modified "2023-10-03" @default.
- W4308175635 title "Non-Modulator Therapies" @default.
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- W4308175635 doi "https://doi.org/10.1016/j.ccm.2022.06.011" @default.
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