r(GGGGCC)n binder 1a

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Chemical Structure : r(GGGGCC)n binder 1a

Catalog No.: PC-61243Not For Human Use, Lab Use Only.

r(GGGGCC)n binder 1a is a bioactive, specific small molecule that targets RNA of the expanded repeat (r(GGGGCC)exp) with Kd of 9.7 uM.

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Purity & Documentation Purity: >98% (HPLC) Select Batch:

Biological Activity

r(GGGGCC)n binder 1a is a bioactive, specific small molecule that targets RNA of the expanded repeat (r(GGGGCC)exp) with Kd of 9.7 uM.
r(GGGGCC)n binder 1a significantly decreases poly(GP) of 10, 18 and 47% at 25, 50 or 100 uM, respectively, in (GGGGCC)66-expressing cells.
r(GGGGCC)n binder 1a significantly inhibits RAN translation and foci formation in cultured cells expressing r(GGGGCC)66 and neurons transdifferentiated from fibroblasts of repeat expansion carriers.

Physicochemical Properties

M.Wt	374.507
Formula	C24H28N3O
Appearance	Solid
CAS No.
Storage	Solide Powder -20 °C 12 Months; 4°C 6 Months	In Solvent -80 °C 6 Months; -20°C 6 Months
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Solubility	10 mM in DMSO
Chemical Name/SMILES	9-hydroxy-5,11-dimethyl-2-(2-(piperidin-1-yl)ethyl)-6H-pyrido[4,3-b]carbazol-2-ium

References

1. Su Z, et al. Neuron. 2014 Sep 3;83(5):1043-50.

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