D-156844 (Compound 11a) hydrochloride is an SMN2 promoter activator with an EC50 of 4 nM. It enhances mRNA expression of the mouse SMN in NSC-34 cells and the human SMN2 promoter in fibroblasts from patients with severe type I spinal muscular atrophy (SMA), along with increasing full-length human SMN protein expression. D-156844 hydrochloride can overcome DHFR inhibition and is used in SMA research.
Target:
DHFR|||DNA/RNA Synthesis
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