RECTAS，50MG，SML4094-50MG，Sigma

Biochem/physiol Actions

Orally active, CDC2-like kinase 1 (CLK1) allosteric activator that enhances promotes CLK1/SRSF5 pathway-mediated pre-mRNA splicing events.
RECTAS is an orally active, CDC2-like kinase 1 (CLK1) allosteric activator that enhances SRSF6 c-terminal RS domain phosphorylation by CLK1, thereby promoting CLK1/SRSF5 pathway-mediated pre-mRNA splicing events. RECTAS restores IKBKAP-familial dysautonomia (FD) exon 20 inclusion in cultured cells (2-10 μM; FD patient iPSC-SNs & murine neuro 2A cells) and in IKBKAP-FD transgenic mice in vivo (two p.o. doses of 300 or 400 mg/kg 4h apart). RECTAS suppresses MC38 tumor growth (by 37/47% at 10/25 μM at the end of 33-day treatment via daily intratumoral injection) by reducing aberrant splicing events and enhancing the splice-neoantigens production.