Article
Future therapies for cystic fibrosis.
Nature communications - 8 Feb 2023
Allen Lucy, Allen Lorna, Carr Siobhan B, Davies Gwyneth, Downey Damian, Egan Marie, Forton Julian T, Gray Robert, Haworth Charles, Horsley Alexander, Smyth Alan R, Southern Kevin W, Davies Jane C
Abstract excerpt
We are currently witnessing transformative change for people with cystic fibrosis with the introduction of small molecule, mutation-specific drugs capable of restoring function of the defective protein, cystic fibrosis transmembrane conductance regulator (CFTR). However, despite being a single gene disorder, there are multiple cystic fibrosis-causing genetic variants; mutation-specific drugs are not suitable for...
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