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.
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