RAN Translation Inhibitor; N 1PC X 10MG

Biochem/physiol Actions

Reversible: yes

Primary Target(r(GGGGCC)exp

Cell permeable: yes

General description

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.

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).

Legal Information

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

Other Notes

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

Packaging

Packaged under inert gas

10 mg in Glass bottle

Physical form

Supplied as HBr salt.

Reconstitution

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

Warning

Toxicity: Standard Handling (A)