CFTR
信号通路图
研究领域
抑制剂选择性比较
CFTR产品
| 产品目录 | 产品描述 | 文献引用 | 实验数据 |
|---|---|---|---|
| S6003 |
Ataluren (PTC124)Ataluren (PTC124)选择性诱导核糖体转录通读，但不影响正常的终止密码子，在HEK293细胞中EC50为0.1 μM，可以治疗nonsense mutations (如CFTR无义突变引起的CF)造成的家族遗传性疾病。 Phase 3。 |
Effect of systemic ataluren treatment on mice with the Pax6Sey+/- phenotype. The black arrowhead indicates the lenticular stalk; the black arrow indicates the cornea; and the asterisk indicates the ciliary margin. WT, Pax6+/+; Mt, Pax6Sey/+; L, lens; r, retina. Original magnification, x5.
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| S7139 |
CFTRinh-172CFTRinh-172是电势差不依赖性的选择性CFTR抑制剂，Ki为300 nM，对于MDR1，ATP敏感性钾离子通道或者一系列的转运蛋白没有效果。 |
CFTR inhibitor CFTR_inh172 leads to PGCs disorder in early zebrafish embryo. Analysis of localization of nanos1/vasa positive cells in embryos by WISH at 50% Epiboly stage with top view.
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| S7329 |
IOWH032IOWH032是一种合成的CFTR抑制剂，在CHO-CFTR细胞测定中， IC50为1.01 μM。 Phase 2。 |
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| S8094 |
GlyH-101GlyH-101是一种选择性且可逆的CFTR抑制剂，其Ki为4.3 μM。 |
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| S1144 |
Ivacaftor (VX-770)Ivacaftor (VX-770)是一种CFTR的选择性增强剂，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。 |
(A) CFTR Western blot of normal (NL) and CF HBE cultures treated with VX-809 (5 uM) ± VX-770 (5 uM) for 48 hours. “*” indicates the mature, complex glycosylated form of CFTR, band C; “•” indicates the immature band B. (B) Turnover of rescued ΔF508 in BHK-21 cells. ΔF508 was rescued at 27°C in the presence of VX-809 ± VX-770 for 24 hours. After adding cycloheximide (200 ug/ml, 37°C), cells were lysed at the indicated times and analyzed by Western blotting.
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| S8698 |
GLPG1837GLPG1837是新型的CFTR增效剂，对F508del的EC50为3 nM。相较于Ivacaftor，它对具有III类突变的CFTR突变体具有增强效应。 |
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| S1565 |
VX-809 (Lumacaftor)VX-809 (Lumacaftor)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。Phase 3。 |
△F508CFTR diffusion is increased by different correction mechanisms. A, example trajectories from MDCK cell lines for wtCFTR (black) and △F508CFTR rescued by VX-809 treatment in the absence (red) and presence (blue ) of mCh-CFTR-C. B, summary graph for wtCFTR, r△F508CFTR and CFTR△PDZ diffusion in VX-809-treated and control MDCK cell lines. C, example trajectories for MDCK cells transiently expressing 3FLAG-tagged wtCFTR(black), △F508CFTR rescued by VX-809 (red), CFTR△PDZ ( blue ), and △F508CFTR△PDZ rescued by VX-809 (green). D, summary graph for CFTR dif- fusion in VX-809-treated MDCK transiently transfected with 3FLAG-tagged CFTR constructs. E, example trajectories from MDCK cell lines stably expressing wtCFTR ( left ) and △F508CFTR ( right ) treated with thapsigargin ( black)or CoPo-22 (red) or transiently expressing GFP-GRASP55 (blue ). F, graphs for △F508CFTR ( top ) and wtCFTR (bottom) diffusion in MDCK cell lines treated with thapsigargin or CoPo-22 or transiently expressing GFP-GRASP55. For reference, median and interquartile values for wtCFTR diffusive range are shown by dashed lines(bar 1, top ). G, GFP-GRASP55 is Golgi-localized by immunocytochemical analysis (scale bar, 10 μm). Scale bar in A (500 nm) refers to all trajectories. In panels Band D, statistically significant differences in populations are shown (*, p<0.001). SPT data for MDCK cell lines was derived from 140 –788 trajectories and for transiently transfected MDCK cells from 131–298 trajectories. |
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| S7059 |
Tezacaftor (VX-661)Tezacaftor (VX-661)是第二个F508del CFTR校正药物，协助CFTR蛋白到达细胞表面。Phase 2。 |
(B)ΔISC response to forskolin observed in VX-661-treated CF HBE cells (*P = 0.0021, VX661 vs. vehicle) was prevented by chronic VX-770 treatment and significantly different from VX-661-treated cells (#P=0.0126, VX661 vs. VX661+VX770). (C) The response to CFTR inh-172 that was observed in VX-661-treated cells (*P = 0.0111, VX661 vs. vehicle) was significantly decreased in VX809+VX770 treated cells (#P = 0.0038, VX661 vs. VX661+VX770). Primary CF HBE cultures (ΔF508/ΔF508) were derived from 3 different patients, and 3-4 replicates were performed per patient.
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| S8795New |
FDL169FDL169是CFTR校正剂，用于修复功能缺陷的CFTR蛋白。 |
| 产品目录 | 产品描述 | 文献引用 | 实验数据 |
|---|---|---|---|
| S6003 |
Ataluren (PTC124)Ataluren (PTC124)选择性诱导核糖体转录通读，但不影响正常的终止密码子，在HEK293细胞中EC50为0.1 μM，可以治疗nonsense mutations (如CFTR无义突变引起的CF)造成的家族遗传性疾病。 Phase 3。 |
Effect of systemic ataluren treatment on mice with the Pax6Sey+/- phenotype. The black arrowhead indicates the lenticular stalk; the black arrow indicates the cornea; and the asterisk indicates the ciliary margin. WT, Pax6+/+; Mt, Pax6Sey/+; L, lens; r, retina. Original magnification, x5.
|
|
| S7139 |
CFTRinh-172CFTRinh-172是电势差不依赖性的选择性CFTR抑制剂，Ki为300 nM，对于MDR1，ATP敏感性钾离子通道或者一系列的转运蛋白没有效果。 |
CFTR inhibitor CFTR_inh172 leads to PGCs disorder in early zebrafish embryo. Analysis of localization of nanos1/vasa positive cells in embryos by WISH at 50% Epiboly stage with top view.
|
|
| S7329 |
IOWH032IOWH032是一种合成的CFTR抑制剂，在CHO-CFTR细胞测定中， IC50为1.01 μM。 Phase 2。 |
||
| S8094 |
GlyH-101GlyH-101是一种选择性且可逆的CFTR抑制剂，其Ki为4.3 μM。 |
| 产品目录 | 产品描述 | 文献引用 | 实验数据 |
|---|---|---|---|
| S1144 |
Ivacaftor (VX-770)Ivacaftor (VX-770)是一种CFTR的选择性增强剂，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。 |
(A) CFTR Western blot of normal (NL) and CF HBE cultures treated with VX-809 (5 uM) ± VX-770 (5 uM) for 48 hours. “*” indicates the mature, complex glycosylated form of CFTR, band C; “•” indicates the immature band B. (B) Turnover of rescued ΔF508 in BHK-21 cells. ΔF508 was rescued at 27°C in the presence of VX-809 ± VX-770 for 24 hours. After adding cycloheximide (200 ug/ml, 37°C), cells were lysed at the indicated times and analyzed by Western blotting.
|
|
| S8698 |
GLPG1837GLPG1837是新型的CFTR增效剂，对F508del的EC50为3 nM。相较于Ivacaftor，它对具有III类突变的CFTR突变体具有增强效应。 |
| 产品目录 | 产品描述 | 文献引用 | 实验数据 |
|---|---|---|---|
| S1565 |
VX-809 (Lumacaftor)VX-809 (Lumacaftor)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。Phase 3。 |
△F508CFTR diffusion is increased by different correction mechanisms. A, example trajectories from MDCK cell lines for wtCFTR (black) and △F508CFTR rescued by VX-809 treatment in the absence (red) and presence (blue ) of mCh-CFTR-C. B, summary graph for wtCFTR, r△F508CFTR and CFTR△PDZ diffusion in VX-809-treated and control MDCK cell lines. C, example trajectories for MDCK cells transiently expressing 3FLAG-tagged wtCFTR(black), △F508CFTR rescued by VX-809 (red), CFTR△PDZ ( blue ), and △F508CFTR△PDZ rescued by VX-809 (green). D, summary graph for CFTR dif- fusion in VX-809-treated MDCK transiently transfected with 3FLAG-tagged CFTR constructs. E, example trajectories from MDCK cell lines stably expressing wtCFTR ( left ) and △F508CFTR ( right ) treated with thapsigargin ( black)or CoPo-22 (red) or transiently expressing GFP-GRASP55 (blue ). F, graphs for △F508CFTR ( top ) and wtCFTR (bottom) diffusion in MDCK cell lines treated with thapsigargin or CoPo-22 or transiently expressing GFP-GRASP55. For reference, median and interquartile values for wtCFTR diffusive range are shown by dashed lines(bar 1, top ). G, GFP-GRASP55 is Golgi-localized by immunocytochemical analysis (scale bar, 10 μm). Scale bar in A (500 nm) refers to all trajectories. In panels Band D, statistically significant differences in populations are shown (*, p<0.001). SPT data for MDCK cell lines was derived from 140 –788 trajectories and for transiently transfected MDCK cells from 131–298 trajectories. |
|
| S7059 |
Tezacaftor (VX-661)Tezacaftor (VX-661)是第二个F508del CFTR校正药物，协助CFTR蛋白到达细胞表面。Phase 2。 |
(B)ΔISC response to forskolin observed in VX-661-treated CF HBE cells (*P = 0.0021, VX661 vs. vehicle) was prevented by chronic VX-770 treatment and significantly different from VX-661-treated cells (#P=0.0126, VX661 vs. VX661+VX770). (C) The response to CFTR inh-172 that was observed in VX-661-treated cells (*P = 0.0111, VX661 vs. vehicle) was significantly decreased in VX809+VX770 treated cells (#P = 0.0038, VX661 vs. VX661+VX770). Primary CF HBE cultures (ΔF508/ΔF508) were derived from 3 different patients, and 3-4 replicates were performed per patient.
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| S8795New |
FDL169FDL169是CFTR校正剂，用于修复功能缺陷的CFTR蛋白。 |
