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About This Item
Empirical Formula (Hill Notation):
C21H23Cl2N5O
CAS Number:
Molecular Weight:
432.35
UNSPSC Code:
12352200
NACRES:
NA.77
MDL number:
Assay:
≥98% (HPLC)
Form:
powder
Quality Segment
assay
≥98% (HPLC)
form
powder
color
white to beige
solubility
DMSO: 2 mg/mL, clear
storage temp.
2-8°C
SMILES string
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
Biochem/physiol Actions
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).
Storage Class
11 - Combustible Solids
wgk
WGK 3
flash_point_f
Not applicable
flash_point_c
Not applicable
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