RECTAS | IKAP activator | Probechem Biochemicals

Chemical Structure : RECTAS

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RECTAS (IKAP activator RECTAS)

货号: PC-72580Not For Human Use, Lab Use Only.

RECTAS is a small molecule splice modulator that corrects aberrant splicing of familial dysautonomia (FD), promotes exon 20 inclusion of IKBKAP pre-mRNA and expression of IKAP.

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250 mg		Get quote

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生物&药学活性

RECTAS is a small molecule splice modulator that corrects aberrant splicing of familial dysautonomia (FD), promotes exon 20 inclusion of IKBKAP pre-mRNA and expression of IKAP.
RECTAS (2 uM) had an effect equivalent to kinetin at 50 uM in HeLa cells expressing the IKBKAP-FD reporter.
RECTAS promoted Exon 20 inclusion of endogenous IKBKAP pre-mRNA and increased IKAP expression in cells of patients with FD.
RECTAS at 2 uM recovered the level of IKAP to levels of IKAP in the cell line from the FD carrier.
RECTAS specifically restores the inclusion of weak exons whose recognition is suppressed by hnRNP H1/H2.
Familial dysautonomia (FD) is caused by missplicing of the IκB kinase complex-associated protein (IKAP) gene, which results in the skipping of exon 20, especially in neurons.
RECTAS directly interacts with CDC-like kinases (CLKs) and enhances SRSF6 phosphorylation.
RECTAS restored DJ-1 protein and mitochondrial dysfunction in patient-derived fibroblasts as well as dopaminergic neuronal cell loss in mutant midbrain organoids.

物理化学性质&存储条件

分子量	248.67
分子式	C11H9ClN4O
外观性状	Solid
CAS No.
储存条件	固体粉末 -20°C 12 个月; 4°C 6 个月	配置液 -80°C 6 个月; -20°C 6 Month
Shipping
Solubility	10 mM in DMSO
Chemical Name/SMILES	2-chloro-N-(furan-2-ylmethyl)-5H-pyrrolo[3,2-d]pyrimidin-4-amine

参考文献

1. Yoshida M, et al. Proc Natl Acad Sci U S A. 2015 Mar 3;112(9):2764-9.

2. Boussaad I, et al. Sci Transl Med. 2020 Sep 9;12(560):eaau3960.

3. Ajiro M, et al. Nat Commun. 2021 Jul 23;12(1):4507.

RECTAS (IKAP activator RECTAS)

生物&药学活性

物理化学性质&存储条件

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RECTAS (IKAP activator RECTAS)

生物&药学活性

物理化学性质&存储条件

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