CFTR
CFTR Products
Catalog No. | Information | Product Use Citations | Product Validations |
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S6003 |
Ataluren (PTC124)Ataluren (PTC124) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 μM in HEK293 cells, may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused by CFTR nonsense mutation). 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 (CFTR inhibitor 172) is a voltage-independent, selective CFTR inhibitor with Ki of 300 nM, showing no effects on MDR1, ATP-sensitive K+ channels, or a series of other transporters. |
![]() ![]() 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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S3272New |
Steviol (Hydroxydehydrostevic acid)Steviol (Hydroxydehydrostevic acid, Hydroxy Dehydrostevic Acid, NSC 226902), a major metabolite of the sweetening compound stevioside, inhibits CFTR activity, reduces AQP2 expression and promotes AQP2 degradation. |
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S0198New |
PPQ-102PPQ-102 (CFTR Inhibitor IV) is a potent inhibitor of CFTR. PPQ-102 can completely inhibit CFTR chloride current with IC50 of ~90 nM. |
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S7329 |
IOWH032IOWH032 is a synthetic CFTR inhibitor with IC50 of 1.01 μM in CHO-CFTR cell based assays. Phase 2. |
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S8094 |
GlyH-101GlyH-101 is a selective and reversible CFTR inhibitor with Ki of 4.3 μM. |
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S1144 |
Ivacaftor (VX-770)Ivacaftor (VX-770) is a selective potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM in fisher rat thyroid cells, respectively. |
![]() ![]() (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 (ABBV-974) is a novel CFTR potentiator with an EC50 value of 3 nM for F508del, showing enhanced efficacy on CFTR mutants harboring class III mutations compared to Ivacaftor. |
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S1565 |
VX-809 (Lumacaftor)VX-809 (Lumacaftor, VRT 826809) acts to correct CFTR mutations common in cystic fibrosis by increasing mutant CFTR (F508del-CFTR) maturation,EC50 of 0.1 μM in fisher rat thyroid cells. 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) is a second F508del CFTR corrector and is believed to help CFTR protein reach the cell surface. 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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S8535New |
ABBV-2222 (GLPG2222)ABBV-2222 (GLPG2222) is a potent corrector of CFTR for the treatment of Cystic Fibrosis (CF). |
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S8795 |
FDL169FDL169 is a CFTR corrector that is designed to fix and restore the function of the defective CFTR protein. |
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S8851 |
Elexacaftor (VX-445)Elexacaftor (VX-445) is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector.This is a compound which is not chiral purity. |
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S6599 |
KM11060KM11060 is a small molecule that corrects the processing of cystic fibrosis transmembrane conductance regulator (CFTR) proteins bearing the F508 deletion (F508del) mutation. |
Catalog No. | Information | Product Use Citations | Product Validations |
---|---|---|---|
S6003 |
Ataluren (PTC124)Ataluren (PTC124) selectively induces ribosomal read-through of premature but not normal termination codons, with EC50 of 0.1 μM in HEK293 cells, may provide treatment for genetic disorders caused by nonsense mutations (e.g. CF caused by CFTR nonsense mutation). 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 (CFTR inhibitor 172) is a voltage-independent, selective CFTR inhibitor with Ki of 300 nM, showing no effects on MDR1, ATP-sensitive K+ channels, or a series of other transporters. |
![]() ![]() 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.
|
|
S3272New |
Steviol (Hydroxydehydrostevic acid)Steviol (Hydroxydehydrostevic acid, Hydroxy Dehydrostevic Acid, NSC 226902), a major metabolite of the sweetening compound stevioside, inhibits CFTR activity, reduces AQP2 expression and promotes AQP2 degradation. |
||
S0198New |
PPQ-102PPQ-102 (CFTR Inhibitor IV) is a potent inhibitor of CFTR. PPQ-102 can completely inhibit CFTR chloride current with IC50 of ~90 nM. |
||
S7329 |
IOWH032IOWH032 is a synthetic CFTR inhibitor with IC50 of 1.01 μM in CHO-CFTR cell based assays. Phase 2. |
||
S8094 |
GlyH-101GlyH-101 is a selective and reversible CFTR inhibitor with Ki of 4.3 μM. |
Catalog No. | Information | Product Use Citations | Product Validations |
---|---|---|---|
S1144 |
Ivacaftor (VX-770)Ivacaftor (VX-770) is a selective potentiator of CFTR targeting G551D-CFTR and F508del-CFTR with EC50 of 100 nM and 25 nM in fisher rat thyroid cells, respectively. |
![]() ![]() (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 (ABBV-974) is a novel CFTR potentiator with an EC50 value of 3 nM for F508del, showing enhanced efficacy on CFTR mutants harboring class III mutations compared to Ivacaftor. |
Catalog No. | Information | Product Use Citations | Product Validations |
---|---|---|---|
S1565 |
VX-809 (Lumacaftor)VX-809 (Lumacaftor, VRT 826809) acts to correct CFTR mutations common in cystic fibrosis by increasing mutant CFTR (F508del-CFTR) maturation,EC50 of 0.1 μM in fisher rat thyroid cells. 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) is a second F508del CFTR corrector and is believed to help CFTR protein reach the cell surface. 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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S8535New |
ABBV-2222 (GLPG2222)ABBV-2222 (GLPG2222) is a potent corrector of CFTR for the treatment of Cystic Fibrosis (CF). |
||
S8795 |
FDL169FDL169 is a CFTR corrector that is designed to fix and restore the function of the defective CFTR protein. |
||
S8851 |
Elexacaftor (VX-445)Elexacaftor (VX-445) is a next-generation cystic fibrosis transmembrane conductance regulator (CFTR) corrector.This is a compound which is not chiral purity. |
||
S6599 |
KM11060KM11060 is a small molecule that corrects the processing of cystic fibrosis transmembrane conductance regulator (CFTR) proteins bearing the F508 deletion (F508del) mutation. |