ASP7967 hemifumarate
ASP7967 hemifumarate is an orally active ligand of the RNA aptamer AC17-4 with a Kd of 12 nM. ASP7967 hemifumarate binds AC17-4 to regulate aptazyme-based and exon-skipping riboswitches, thereby enabling small-molecule-controlled transgene expression without exogenous protein factors. ASP7967 hemifumarate can be used for the study of synthetic RNA switches, gene expression regulation and AAV-based gene therapy-related expression control.
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- No. CAS: 1246958-42-8
- Fòrmula: C24H20F2N8O4
- Peso molecular:464.43
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Almacenamiento:
Please store the product under the recommended conditions in the Certificate of Analysis.
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Actividad biológica
Descripciòn
IC50 & Target
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AC17-4 aptamer 12 nM (Kd) |
In Vitro
ASP7967 hemifumarate binds the RNA aptamer AC17-4 with a Kd of 12 nM[1].
ASP7967 (5 μM; 2 days after transfection) hemifumarate activates EGFP expression by approximately 10-fold in HEK293 cells expressing the a8-AC17-4-CPP aptazyme riboswitch[1].
ASP7967 (10 μM; 2 days after transfection) hemifumarate mostly saturates the a8-AC17-4-CPP riboswitch response in HEK293 cells, with an EC50 of approximately 2.3 μM and a fully induced ON level of approximately 50% of the empty-vector control[1].
ASP7967 (0-10 μM; 18 h) hemifumarate does not significantly reduce HEK293 cell viability in the MTT assay[1].
ASP7967 (0.1-10 μM; 2 days after transfection) hemifumarate regulates hEPO secretion from HEK293 cells transfected with pAAV-CMV-hEPO-a8c-AC17-4-CPP, at 10 μM, it upregulates hEPO secretion by approximately 10-fold, reaching 29% of the no-riboswitch control construct[1].
ASP7967 hemifumarate triggers EGFP expression via riboswitches with ON/OFF ratios of 114, 58 and 177 respectively in HEK293 cells[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Cell Line:HEK293
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Concentration:10 μM
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Incubation Time:2 days after transfection
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Result:Upregulated hEPO secretion by approximately 10-fold in cells transfected with pAAV-CMV-hEPO-a8c-AC17-4-CPP.
Increased hEPO secretion to 29% of the no-riboswitch control construct.
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Cell Line:HEK293
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Concentration:0, 2, 5, 10 µM
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Incubation Time:2 days after transfection
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Result:Did not significantly reduce HEK293 cell viability in the MTT assay.
Parmacokinetics
| Species | Dose | Route | Tmax |
|---|---|---|---|
| Mice[1] | 100 mg/kg | p.o. | 1 h |
In Vivo
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:7-week-old male BALB/c mice intravenously injected with 3 × 1010 genome copies of AAV8-CMV-hEPO-a8c-AC17-4-CPP per mouse[1]
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Dosage:100 mg/kg
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Administration:Oral administration (p.o.); single administration; 2 weeks after AAV injection
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Result:Upregulated serum hEPO expression in mice.
Increased serum hEPO to approximately 115 mIU/mL at 6-8 h postdosing.
Produced a 7.2-fold increase in serum hEPO over vehicle control.
Serum hEPO subsequently decreased in accordance with the decline of ASP7967 concentration in the liver.
Chemical Information
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No. CAS 1246958-42-8
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Peso molecular 464.43
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Fòrmula C24H20F2N8O4
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SMILES
OC(/C=C/C(O)=O)=O.FC(C=C1)=CC=C1NC2=NC(NC3=CC=C(C=C3)F)=NC(NCC4=NC=CC=N4)=N2.[1/2]
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Envío
Room temperature in continental US; may vary elsewhere.
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Almacenamiento
Please store the product under the recommended conditions in the Certificate of Analysis.
Pureza y Documentación
Referencias
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)
ASP7967 hemifumarate Related Classifications
HY-49538C Related Classifications
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