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Description: IKBKAP pre-mRNA splicing modulator
Chemical Name: 2-Chloro-N-(2-furanylmethyl)-7H-purin-6-amine
Purity: ≥98% (HPLC)
Literature (2)

Biological Activity for RECTAS

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.

Technical Data for RECTAS

M. Wt 249.66
Formula C10H8ClN5O
Storage Store at -20°C
Purity ≥98% (HPLC)
CAS Number 101862-47-9
PubChem ID 5037694
Smiles ClC1=NC=2N=CNC2C(=N1)NCC=3OC=CC3

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.

Solubility Data for RECTAS

Solvent Max Conc. mg/mL Max Conc. mM
DMSO 24.97 100

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 will affect the solvent volumes required to prepare stock solutions.

Select a batch to recalculate based on the batch molecular weight:
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

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References for RECTAS

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

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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

Citations for RECTAS

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