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Submit ReviewRECTAS (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.
分子量 | 249.66 |
公式 | C10H8ClN5O |
储存 | Store at -20°C |
纯度 | ≥98% (HPLC) |
CAS Number | 101862-47-9 |
PubChem ID | 5037694 |
InChI Key | QGUOPVCOXHVPJX-UHFFFAOYSA-N |
Smiles | ClC1=NC=2N=CNC2C(=N1)NCC=3OC=CC3 |
上方提供的技术数据仅供参考。批次相关数据请参见分析证书。
Tocris products are intended for laboratory research use only, unless stated otherwise.
溶剂 | 最高浓度 mg/mL | 最高浓度 mM | |
---|---|---|---|
溶解性 | |||
DMSO | 24.97 | 100 |
以下数据基于产品分子量 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.
浓度/溶剂体积/质量 | 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 |
参考文献是支持产品生物活性的出版物。
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.
关键词: 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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