Lucerastat-NB-DGJ-DataSheet-生命科学试剂-MedChemExpress

Hotline:400-820-3792Inhibitors•ScreeningLibraries•Proteinswww.MedChemELucerastatCat.No.:HY-106392CASNo.:141206-42-0Synonyms:NB-DGJ;N-(n-Butyl)deoxygalactonojirimycin分⼦式:C₁₀H₂₁NO₄分⼦量:219.28作⽤靶点:GlucosylceramideSynthase(GCS)作⽤通路:NeuronalSignaling储存⽅式:-20°C,sealedstorage,awayfrommoisture*Insolvent:-80°C,6months;-20°C,1month(sealed

storage,awayfrommoisture)溶解性数据体外实验H2O:24mg/mL(109.45mM;ultrasonicandwarmingandheatto80°C)DMSO:22mg/mL(100.33mM;ultrasonicandwarmingandheatto80°C)MassSolvent1mg5mg10mgConcentration制备储备液1mM4.5604mL22.8019mL45.6038mL5mM0.9121mL4.5604mL9.1208mL10mM0.4560mL2.2802mL4.5604mL请根据产品在不同溶剂中的溶解度选择合适的溶剂配制储备液；⼀旦配成溶液，请分装保存，避免反复冻融造成的产品失效。储备液的保存⽅式和期限：-80°C,6months;-20°C,1month(sealedstorage,awayfrommoisture)。-80°C储存时，请在6个⽉内使⽤，-20°C储存时，请在1个⽉内使⽤。BIOLOGICALACTIVITY⽣物活性LucerastaMiglustat的半乳糖形式，具有⼝服活性的葡萄糖酰神经酰胺合成酶(GCS)的抑制剂。Lucerastat有⽤于Fabry疾病研究的潜能。体外研究Fabrypatient-derivedfibroblastswiththegenotypesR301G(residual-GalAactivity;20%)R220X(<3%)and1/2MasterofBioactiveMolecules—您⾝边的抑制剂⼤师www.MedChemEW162X(<1%).CellViabilityAssay[2].CellLine:Fabrypatient-derivedfibroblastswiththegenotypesR301G(residual-GalAactivity;20%)R220X(<3%)andW162X(<1%).Concentration:IncubationTime:9days.Result:Dose-dependentlyinhibitedGCS,reducingglucosylceramideandincreasingsphingomyelin.体内研究Lucerastat(1200mg/kg/dayfoodadmix),aGCSinhibitor,reducesGb3intheabsenceofresidual-GalAactivity[2].AnimalModel:Fabrymice(Gla-/0andGla-/-,n=5or6foreachgender)[2].Dosage:1200mg/kg/dayfoodadmix.Administration:Foodadmixfor20weeks.Result:ReducedlipidstorageintwomajororgansaffectedbyFD:meanGb3inthekidneys(-33%,p<0.01).andα-galactose-terminatedglycosphingolipidsinthedorsalrootganglia(-48%,p<0.05).IntheliveroftheFabrymice,meanglucosylceramide(GlcCer(24:0))wasreduced(-59%,p<0.001)inadditiontoGb3(24:1)(-37%,p<0.05)demonstratingsubstratereductionthroughGCSinhibition.REFERENCES[1].SanneJvanderVeen,etal.DevelopmentsintheTreatmentofFabryDisease.JInheritMetabDis.2020Feb21.[2].R.W.D.Welford,etal.Lucerastat,anIminosugarforSubstrateReductionTherapyinFabryDisease:PreclinicalEvidence.McePdfHeightCaution:Producthasnotbeenfullyvalidatedformedicalapplications.Forresear