Hotline:400-820-3792Inhibitors?ScreeningLibraries?Proteinswww.MedChemEBranaplamhydrochlorideCat.No.:HY-19620ACASNo.:1562338-39-9Synonyms:LMI070hydrochloride;NVS-SM1hydrochloride分?式:C??H??ClN?O?分?量:429.94作?靶點(diǎn):DNA/RNASynthesis;PotassiumChannel作?通路:CellCycle/DNADamage;MembraneTransporter/IonChannel儲(chǔ)存?式:4°C,sealedstorage,awayfrommoisture*Insolvent:-80°C,6months;-20°C,1month(sealed

storage,awayfrommoisture)溶解性數(shù)據(jù)體外實(shí)驗(yàn)H2O:200mg/mL(465.18mM;Needultrasonic)MassSolvent1mg5mg10mgConcentration制備儲(chǔ)備液1mM2.3259mL11.6295mL23.2591mL5mM0.4652mL2.3259mL4.6518mL10mM0.2326mL1.1630mL2.3259mL請(qǐng)根據(jù)產(chǎn)品在不同溶劑中的溶解度選擇合適的溶劑配制儲(chǔ)備液；?旦配成溶液，請(qǐng)分裝保存，避免反復(fù)凍融造成的產(chǎn)品失效。儲(chǔ)備液的保存?式和期限：-80°C,6months;-20°C,1month(sealedstorage,awayfrommoisture)。-80°C儲(chǔ)存時(shí)，請(qǐng)?jiān)?個(gè)?內(nèi)使?，-20°C儲(chǔ)存時(shí)，請(qǐng)?jiān)?個(gè)?內(nèi)使?。BIOLOGICALACTIVITY?物活性Branaplam(LMI070;NVS-SM1)hydrochloride?種?服有效和選擇性的SMN2拼接調(diào)節(jié)劑，對(duì)SMN的EC50為20nM。Branaplamhydrochloride抑制hERG，IC50為6.3μM。Branaplamhydrochloride在嚴(yán)重的脊髓性肌萎縮癥(SMA)??模型中可提?全長(zhǎng)SMN蛋?并延長(zhǎng)其?存期。體外研究Branaplam(LMI070;NVS-SM1)hydrochloridetreatmentinduceschangesinthelevelsof175genesin1/2MasterofBioactiveMolecules—您?邊的抑制劑?師www.MedChemEhumanfibroblasts[1].體內(nèi)研究Branaplam(LMI070;NVS-SM1;3,10,30mg/kg;oral)hydrochlorideproducesdose-dependentelevationsofSMN2-FLtranscriptandSMNproteininbrainandspinalcord[1].Branaplam(1mg/kgofIV;3mg/kgofPO)hydrochloridehasaCLof25mL/min/kgandanAUCof3.03μM?h[2].AsingleBranaplam(oral;30mg/kg)hydrochlorideresultsinsignificantanddurableSMNproteinelevationinbrainforupto160hoursinC/+mice[1].Branaplam(oral;0.03,0.1,0.3,1,3mg/kg)hydrochlorideimprovesbodyweightandextendeslifespaninnSMNΔ7mice[1].戶(hù)使?本產(chǎn)品發(fā)表的科研?獻(xiàn)?Nature.2021Aug;596(7871):291-295.?NucleicAcidsRes.2021Sep7;49(15):8462-8470.?bioRxiv.2020Jun.?bioRxiv.2020Feb.Seemorecustomervalidationsonwww.MedChemEREFERENCES[1].PalacinoJ,etal.SMN2splicemodulatorsenhanceU1-pre-mRNAassociationandrescueSMAmice.NatChemBiol.2015Jul;11(7):511-517.[2].CheungAK,etal.DiscoveryofSmallMoleculeSplicingModulatorsofSurvivalMotorNeuron-2(SMN2)fortheTreatmentofSpinalMuscularAtrophy(SMA).JMedChem.2018Dec27;61(24):11021-11036.McePdfHeightCaution:Producthasnotbeenfullyvalidatedformedicalapplications.Forresearc