A cell permeable indole fused isoquinolinium salt that binds to the expanded repeat (r(GGGGCC)exp (K_d = 9.7 µM) 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 µM; 47% at to 100 µM). Also shown to inhibit RAN translation and foci formation in (GGGGCC)exp-expressing C9ORF72+ iNeurons (~ 2 µM).

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.

Cell permeable: yes

Primary Target
(r(GGGGCC)exp

Reversible: yes

Packaged under inert gas

Supplied as HBr salt.

Following reconstitution, aliquot and freeze (-20°C). Stock solutions are stable for up to 3 months at -20°C.

Su, Z., et al. 2014. Neuron.83, 1043.

CALBIOCHEM is a registered trademark of Merck KGaA, Darmstadt, Germany

Toxicity: Standard Handling (A)