RECTAS

Product 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.

Solubility

Preparing Stock Solutions for RECTAS

The following data is based on the product molecular weight 249.66

Batch specific molecular weights may vary from batch to batch due to the degree of hydration, which all affect the solvent volumes required to prepare stock solutions.

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Concentration / Solvent Volume / Mass	1 mg	5 mg	10 mg
1 mM	4.01 mL	20.03 mL	40.05 mL
5 mM	0.80 mL	4.01 mL	8.01 mL
10 mM	0.40 mL	2.00 mL	4.01 mL
50 mM	0.08 mL	0.40 mL	0.80 mL

Calculators

Background References

References are publications that support the biological activity of the product.

• Yoshida Rectifier of aberrant mRNA splicing recovers tRNA modification in familial dysautonomia. Proc.Natl.Acad.Sci U.S.A. 2015 PMID: 25675486
• Boussaad A patient-based model of RNA mis-splicing uncovers treatment targets in Parkinson's disease. Sci.Transl.Med. 2020 PMID: 32908004
• Ajiro Therapeutic manipulation of IKBKAP mis-splicing with a small molecule to cure familial dysautonomia. Nat.Commun. 2021 PMID: 34301951