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《rnai治疗的大时代（the great age of rnai therapy）》由会员上传分享，免费在线阅读，更多相关内容在教育资源-天天文库。
1、RNAi治疗的大时代（ThegreatageofRNAitherapy）RNAinterference(RNAi)isamechanismoftranscriptionalsilencethatexistsinnature.Sixteenyearsago,scientistsbegantofocusonthepotentialofthismechanismfortreatment,especiallyfordiseasesthatareoftenineffectiveinconventionaldrugs,suchascancer.Butformorethanadecade,RNAi
2、treatmentshavebeenaroller-coasterridetoclinicalapplications.AndrewFireandCraigMello,whowontheNobelPrizeforphysiologyandmedicinein2006,firstrevealedtheRNAiphenomenonin1998innematodeworms.Sincethen,RNAimethodbecauseofitssimpleoperationandgoodexperimentaleffectSoonbecomethemaintechnologyofbasicsci
3、entificresearch,researchersaroundtheworldbegantousethismethodtosilencegenesinthecellandanimalmodels.Atthesametime,moreandmorescientistsarebecominginterestedinRNAitreatment.Higher,anddoesnotneedspecialbindingprotein,candirectlybeusedinmonoclonalantibodycannotreachthetarget,preventingpathogenicpr
4、oteinproducedsomegenetranslationdirectly,borninsourceblockthedisease,toavoidtheuseofsmallmoleculedrugsorotherbiologicalagentsUnicomroaddownstreamtotreatdiseaseatthemolecularlevel.DespitetheadvantagesofRNAitreatment,therearestillscholarswaryofRNAitreatment.Pharmaceuticalcompanieshavestartedtobur
5、nmoneyinthisareainanefforttogainacommandingheightsintechnology.Rationalboom"phase.Aninseverallargepharmaceuticalcompaniesarenotundertheconditionofthetechnicalobstacleshaveinvestedbillionsofdollars,andbegantolookatsomeoftheexistingtechnologyresearchanddevelopmenttoRNAitherapy,particularlythosege
6、netictargets,butthereisnodrugdisease.However,theearlyclinicalresultsareverydisappointing,RNAitherapydidnotachievetheexpectedeffect.Moststrikingly,thewetmaculardegenerationRNAi,developedbyMiamipharmaceuticalcompanyOPKOin2009,hadtobeabandonedinclinicalphaseIIItrialsbecauseofpoorresults.Hisdrug,ba
7、sedonRNAi,iseithernoteffectiveorhasasevereautoimmuneresponse,failingtopassclinicaltrials.RNAitherapybeginstoenterastageof"doubtanddespair".Thepharmaceuticalgiants,whohadbeenburningcashbefore,begantoaxetheRNAiprojectinthefaceofturm
