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Description

A cell permeable indole fused isoquinolinium salt that binds to the expanded repeat (r(GGGGCC)exp (Kd = 9.7 µ,M) with modest selectivity. This binding is dependent upon the ionic strength and appears to be significantly weaker in the presence of Na+ and K+. Inhibits repeat-associated non-ATG (RAN) translation and foci Formation in r(GGGGCC)66-expressing HEK293 cells and reduces the levels of poly(GP) and poly(GA) proteins (10% at 25 µ,M, 47% at to 100 µ,M). Also shown to inhibit RAN translation and foci formation in (GGGGCC)exp-expressing C9ORF72+ iNeurons (~ 2 µ,M).Please note that the molecular weight for this compound is batch-specific due to variable water content. Please refer to the vial label or the certificate of analysis for the batch-specific molecular weight. The molecular weight provided represents the baseline molecular weight without water., A cell permeable indole fused isoquinolinium salt that binds to the expanded repeat (r(GGGGCC)exp (Kd = 9.7 µ,M) with modest selectivity. This binding is dependent upon the ionic strength and appears to be significantly weaker in the presence of Na+ and K+. Inhibits repeat-associated non-ATG (RAN) translation and foci Formation in r(GGGGCC)66-expressing HEK293 cells and reduces the levels of poly(GP) and poly(GA) proteins (10% at 25 µ,M, 47% at to 100 µ,M). Also shown to inhibit RAN translation and foci formation in (GGGGCC)exp-expressing C9ORF72+ iNeurons (~ 2 µ,M).