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Partial rescue of F508del-cystic fibrosis transmembrane conductance regulator channel gating with modest improvement of protein processing, but not stability, by a dual-acting small molecule.
Liu J, Bihler H, Farinha CM, Awatade NT, Romão AM, Mercadante D, Cheng Y, Musisi I, Jantarajit W, Wang Y, Cai Z, Amaral MD, Mense M, Sheppard DN. Liu J, et al. Among authors: wang y. Br J Pharmacol. 2018 Apr;175(7):1017-1038. doi: 10.1111/bph.14141. Epub 2018 Feb 22. Br J Pharmacol. 2018. PMID: 29318594 Free PMC article.
A topological switch in CFTR modulates channel activity and sensitivity to unfolding.
Scholl D, Sigoillot M, Overtus M, Martinez RC, Martens C, Wang Y, Pardon E, Laeremans T, Garcia-Pino A, Steyaert J, Sheppard DN, Hendrix J, Govaerts C. Scholl D, et al. Among authors: wang y. Nat Chem Biol. 2021 Sep;17(9):989-997. doi: 10.1038/s41589-021-00844-0. Epub 2021 Aug 2. Nat Chem Biol. 2021. PMID: 34341587
CFTR bearing variant p.Phe312del exhibits function inconsistent with phenotype and negligible response to ivacaftor.
Raraigh KS, Paul KC, Goralski JL, Worthington EN, Faino AV, Sciortino S, Wang Y, Aksit MA, Ling H, Osorio DL, Onchiri FM, Patel SU, Merlo CA, Montemayor K, Gibson RL, West NE, Thakerar A, Bridges RJ, Sheppard DN, Sharma N, Cutting GR. Raraigh KS, et al. Among authors: wang y. JCI Insight. 2022 Mar 22;7(6):e148841. doi: 10.1172/jci.insight.148841. JCI Insight. 2022. PMID: 35315358 Free PMC article.
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