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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.
|Storage||Store at -20°C|
The technical data provided above is for guidance only. For batch specific data refer to the Certificate of Analysis.
Tocris products are intended for laboratory research use only, unless stated otherwise.
|Solvent||Max Conc. mg/mL||Max Conc. mM|
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 will affect the solvent volumes required to prepare stock solutions.
|Concentration / Solvent Volume / Mass||1 mg||5 mg||10 mg|
|1 mM||4.01 mL||20.03 mL||40.05 mL|
|5 mM||0.8 mL||4.01 mL||8.01 mL|
|10 mM||0.4 mL||2 mL||4.01 mL|
|50 mM||0.08 mL||0.4 mL||0.8 mL|
References are publications that support the biological activity of the product.
Ajiro et al (2021) Therapeutic manipulation of IKBKAP mis-splicing with a small molecule to cure familial dysautonomia. Nat.Commun. 12 4507 PMID: 34301951
Yoshida et al (2015) Rectifier of aberrant mRNA splicing recovers tRNA modification in familial dysautonomia. Proc.Natl.Acad.Sci U.S.A. 112 2764 PMID: 25675486
Boussaad et al (2020) A patient-based model of RNA mis-splicing uncovers treatment targets in Parkinson's disease. Sci.Transl.Med. 12 eaau3960 PMID: 32908004
If you know of a relevant reference for RECTAS, please let us know.
Keywords: RECTAS, RECTAS supplier, IKBKAP, splicing, modulator, modulates, familial, dysautonomia, FD, brain, penetrant, orally, bioavailable, PARK7, DJ-1, parkinsons, disease, DNA,, RNA, and, Protein, Synthesis, Polymerase, 7679, Tocris Bioscience
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