RECTAS

Pricing Availability   Qty
说明: IKBKAP pre-mRNA splicing modulator
化学名: 2-Chloro-N-(2-furanylmethyl)-7H-purin-6-amine
纯度: ≥98% (HPLC)
说明书
引用文献
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文献 (2)

生物活性 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.

技术数据 for RECTAS

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.

溶解性数据 for RECTAS

溶剂 最高浓度 mg/mL 最高浓度 mM
溶解性
DMSO 24.97 100

制备储备液 for RECTAS

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.

选择批次从而根据批次分子量重新计算:
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

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参考文献 for RECTAS

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


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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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