ASP7967 hemifumarate 

ASP7967 hemifumarate is an orally active ligand of the RNA aptamer AC17-4 with a K_d 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^[1].

ASP7967 hemifumarate binds the RNA aptamer AC17-4 with a K_d 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.

ASP7967 (100 mg/kg; p.o.; single administration, 2 weeks after AAV injection; blood collected at 2,4,6,8,24 h postdose) hemifumarate upregulates serum hEPO in BALB/c mice injected with 3 × 10¹⁰ GC AAV8-CMV-hEPO-a8c-AC17-4-CPP; serum hEPO peaks at ~115 mIU/mL (6-8 h) with a 7.2-fold elevation vs vehicle, while no hEPO change occurs in mice receiving control AAV8-CMV-hEPO^[1].

MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.

464.43

C₂₄H₂₀F₂N₈O₄

1246958-42-8

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]

Room temperature in continental US; may vary elsewhere.

Please store the product under the recommended conditions in the Certificate of Analysis.

• [1]. Fukunaga K et al. Small-Molecule Aptamer for Regulating RNA Functions in Mammalian Cells and Animals. J Am Chem Soc. 2023 Apr 12;145(14):7820-7828.