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Emerging gene therapies for cystic fibrosis include inhaled delivery of CFTR mRNA in lipid nanoparticles or CFTR cDNA in viral vectors as mutation-agnostic approaches to restore
Nov 1, 2023 · CFTR encodes for an ion channel, CFTR, that is involved in regulation of the water–electrolyte balance on the surface of many organ systems, including the upper and lower airways, intestine,...
Sep 30, 2023 · A phase 3, open-label, 96-week trial to study the safety, tolerability, and efficacy of tezacaftor/ivacaftor in children ≥ 6 years of age homozygous for F508del or heterozygous for F508del and a residual function CFTR variant
Feb 8, 2023 · Those patients with gene variants responsive to CFTR modulators may have found these therapies to be transformational; research is now focusing on safely reducing the burden of symptom-directed...
May 6, 2021 · As we look to the future, when most children and adults with cystic fibrosis will be eligible for treatment with highly effective CFTR modulator therapy, we will rely on data from international multicentre studies to further elucidate the effects of these drugs beyond lung function.
Oct 30, 2021 · The CF gene encodes for a protein the cystic fibrosis transmembrance conductance regulator (CFTR) which is a protein chloride channel that belongs to the family of adenosine triphosphate (ATP)-binding cassette (ABC) transporters.
- Jin-A Lee, Alex Cho, Elena N. Huang, Yiming Xu, Henry Quach, Jim Hu, Amy P. Wong
- 10.1186/s12967-021-03099-4
- 2021
- J Transl Med. 2021; 19: 452.
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Mary Dwight. Bruce Marshall, MD. Advances in treatment and care have transformed cystic fibrosis (CF). Once considered a pediatric disease, today more than half of all people with CF are over the age of 18 years.
