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Tocris Bioscience™ RECTAS
IKBKAP pre-mRNA splicing modulator
Brand: Tocris Bioscience™ 7679/50
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Description
RECTAS (rectifier of aberrant splicing) is a pre-mRNA splicing modulator of the IKBKAP (inhibitor of κ light polypeptide gene enhancer in B-cells, kinase complex associated protein) gene. It corrects aberrant IKBKAP splicing in vitro in cellular models of familial dysautonomia and in vivo in transgenic mice. In combination with Phenylbutyric acid (Cat. No. 2682), RECTAS rescues PARK7 aberrant splicing and neuronal cell loss in a midbrain organoid model of familial Parkinson's disease. Orally bioavailable and brain penetrant.Specifications
2-Chloro-N-(2-furanylmethyl)-7 H-purin-6-amine | |
50 mg | |
C10H8ClN5O |
101862-47-9 | |
DNA, RNA and Protein Synthesis Compounds | |
0.98 |
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