使用 'F508del-CFTR' 作为标签的产品
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CFTR modulator
VX-809 (Lumacaftor)是用于治疗囊性纤维化（CF）的第二种口服研究候选化合物，通过促进突变型CFTR(F508del-CFTR)的成熟，从而纠正囊性纤维症中常见的CFTR突变，在fisher大鼠甲状腺细胞中EC50为0.1 μM。
了解更多
- Kusumika Saha, .et al. Pharmacological chaperone-rescued cystic fibrosis CFTR-F508del mutant overcomes PRAF2-gated access to endoplasmic reticulum exit sites, Cell Mol Life Sci, 2022, Sep 27;79(10):530 PMID: 36167862
- Heledd H Jarosz-Griffiths, .et al. Different CFTR Modulator Combinations Downregulate Inflammation Differently in Cystic Fibrosis, Elife, 2020, Mar 2;9:e54556 PMID: 32118580
- Okiyoneda T, .et al. Chaperone-Independent Peripheral Quality Control of CFTR by RFFL E3 Ligase, Dev Cell, 2018, Mar 26;44(6):694-708.e7 PMID: 29503157
- Junko Kido, .et al. Cystic Fibrosis Transmembrane Conductance Regulator Reduces Microtubule-Dependent Campylobacter jejuni Invasion, Infect Immun, 2017, Oct; 85(10): e00311-17 PMID: 28784926
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CFTR activator
VX-770 (Ivacaftor)被称为CFTR增强剂，靶向作用于G551D-CFTR和F508del-CFTR，在fisher大鼠甲状腺细胞中EC50分别为100 nM和25 nM。
了解更多
- Okiyoneda T, .et al. Chaperone-Independent Peripheral Quality Control of CFTR by RFFL E3 Ligase, Dev Cell, 2018, Mar 26;44(6):694-708.e7 PMID: 29503157
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CFTR corrector
VX-661是另一种正在开发中的用于治疗囊性纤维化的囊性纤维化跨膜电导调节器（CFTR）校正器。
了解更多
- Kusumika Saha, .et al. Pharmacological chaperone-rescued cystic fibrosis CFTR-F508del mutant overcomes PRAF2-gated access to endoplasmic reticulum exit sites, Cell Mol Life Sci, 2022, Sep 27;79(10):530 PMID: 36167862
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