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1、NucleicAcidsResearchAdvanceAccesspublishedMarch23,2015NucleicAcidsResearch,20151doi:10.1093/nar/gkv226SURVEYANDSUMMARYAdvancesinCRISPR-Cas9genomeengineering:lessonslearnedfromRNAinterferenceRodolpheBarrangou1,†,AmandaBirmingham2,†,StefanWiemann3,RoderickL.Beijersbergen4,VeitHornung5andAnjavanBraban
2、tSmith2,*1DepartmentofFood,BioprocessingandNutritionSciences,NorthCarolinaStateUniversity,Raleigh,NC27695,USA,2Dharmacon,partofGEHealthcare,Lafayette,CO80026,USA,3DivisionofMolecularGenomeAnalysis,andGenomic&ProteomicsCoreFacility,GermanCancerResearchCenter,69120Heidelberg,Germany,4TheDownloadedfro
3、mNetherlandsCancerInstitute,1066CXAmsterdam,TheNetherlandsand5InstituteofMolecularMedicine,UniversityHospital,UniversityofBonn,53128Bonn,GermanyReceivedJanuary15,2015;RevisedMarch4,2015;AcceptedMarch5,2015http://nar.oxfordjournals.org/ABSTRACTgenomescaleinhighereukaryotes(1).Inthelast24months,anoth
4、ergenemodulationtechnique,ClusteredRegularlyThediscoverythatthemachineryoftheClusteredInterspacedShortPalindromicRepeats(CRISPR)-Cas9RegularlyInterspacedShortPalindromicRepeatsgenomeengineering(referredtoasCRISPR-Cas9),has(CRISPR)-Cas9bacterialimmunesystemcanbere-emerged;inthatremarkablybriefwindow
5、,thisapproachpurposedtoeasilycreatedeletions,insertionsandhasproventobeapowerfultoolforstudyingindividualreplacementsinthemammaliangenomehasrevo-genefunction,performinggenome-widescreens,creatingatSouthernMedicalUniversityonJune30,2015lutionizedthefieldofgenomeengineeringandre-diseasemodelsandperhap
6、sdevelopingtherapeuticagentsinvigoratedthefieldofgenetherapy.Manyparal-(2).Theselightningadvanceshavelargelyfollowedthepathlelshavebeendrawnbetweenthenewlydiscov-blazedbyRNAistudiesandwearguethatfurtherleverageeredCRISPR-Cas9systemandtheRNAinterfer-istobegainedbyexaminingrelevantsuccessesandfailures
7、inthelast14yearsofRNAi.ence(RNAi)pathwayintermsoftheirutilityforRNAiandCRISPR-Cas9havemanyclearsimilarities.understandingandinterrogatinggenefunctioninIndeed,themechanismsofbothusesmallRNAswithanmammaliance