おすすめの製品
品質水準
アッセイ
≥98% (HPLC)
フォーム
powder
色
white to beige
溶解性
DMSO: 2 mg/mL, clear
保管温度
2-8°C
SMILES記法
NC1=NC(N)=C2C(OCC3CCN(CC3)CC4=C(C=CC=C4Cl)Cl)=CC=CC2=N1
InChI
1S/C21H23Cl2N5O/c22-15-3-1-4-16(23)14(15)11-28-9-7-13(8-10-28)12-29-18-6-2-5-17-19(18)20(24)27-21(25)26-17/h1-6,13H,7-12H2,(H4,24,25,26,27)
InChI Key
MNLHFGXIUJNDAF-UHFFFAOYSA-N
生物化学的／生理学的作用
Brain-penetrant, potent mRNA-decapping enzyme DcpS (m7GpppX diphosphatase) inhibitor within in vivo efficacy in a murine spinal muscular atrophy (SMA) model.
RG3039 is a brain-penetrant and potent inhibitor against the scavenger mRNA-decapping enzyme DcpS (m7GpppX diphosphatase) in vitro (mouse DcpS IC50 = 3.4 nM) and in mice in vivo (∼90% & ∼80% inhibition of brain DcpS, respectively, 2 h & 72 h post last 3 mg/kg daily ip. from P1 to P10). In a murine severe spinal muscular atrophy (SMA) model, RG3039 (10-20 mg/kg/d ip. from P1 till death) increases SMAΔ7 mice survival (by 26%) and motor function with a ∼50% increase of VGLUT1 synapses number on L3-L5 motor neurons (33.7/WT mice at P13 vs. 17.2/SMA mice without 26.1/SMA mice with RG3039 treatment).
保管分類コード
11 - Combustible Solids
WGK
WGK 3
引火点(°F)
Not applicable
引火点(℃)
Not applicable
適用法令
試験研究用途を考慮した関連法令を主に挙げております。化学物質以外については、一部の情報のみ提供しています。 製品を安全かつ合法的に使用することは、使用者の義務です。最新情報により修正される場合があります。WEBの反映には時間を要することがあるため、適宜SDSをご参照ください。
Jan Code
SML3071-5MG:
SML3071-VAR:
SML3071-BULK:
SML3071-25MG:
最新バージョンのいずれかを選択してください：
Alyssa N Calder et al.
Journal of medicinal chemistry, 59(22), 10067-10083 (2016-08-05)
Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease resulting from pathologically low levels of survival motor neuron (SMN) protein. The majority of mRNA from the SMN2 allele undergoes alternative splicing and excludes critical codons, causing an SMN protein
Takuji Yamauchi et al.
Cancer cell, 33(3), 386-400 (2018-02-27)
To identify novel targets for acute myeloid leukemia (AML) therapy, we performed genome-wide CRISPR-Cas9 screening using AML cell lines, followed by a second screen in vivo. Here, we show that the mRNA decapping enzyme scavenger (DCPS) gene is essential for AML
Rocky G Gogliotti et al.
Human molecular genetics, 22(20), 4084-4101 (2013-06-06)
Spinal muscular atrophy (SMA) is caused by insufficient levels of the survival motor neuron (SMN) protein due to the functional loss of the SMN1 gene and the inability of its paralog, SMN2, to fully compensate due to reduced exon 7
Blazej A Wojtczak et al.
Journal of the American Chemical Society, 140(18), 5987-5999 (2018-04-21)
The 5' cap consists of 7-methylguanosine (m7G) linked by a 5'-5'-triphosphate bridge to messenger RNA (mRNA) and acts as the master regulator of mRNA turnover and translation initiation in eukaryotes. Cap analogues that influence mRNA translation and turnover (either as
Jonathan J Cherry et al.
PloS one, 12(9), e0185079-e0185079 (2017-09-26)
C5-substituted 2,4-diaminoquinazoline inhibitors of the decapping scavenger enzyme DcpS (DAQ-DcpSi) have been developed for the treatment of spinal muscular atrophy (SMA), which is caused by genetic deficiency in the Survival Motor Neuron (SMN) protein. These compounds are claimed to act
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