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《基因治疗Gene Therapy.ppt》由会员上传分享，免费在线阅读，更多相关内容在行业资料-天天文库。
1、GeneTherapy■BackgroundCancerCCVD:Cardio-Cerebrovascular DiseasesViralDiseaseGenetic Disease:morethan2000diseasesCNS:Central Nervous System DiseaseImmune DiseasesDiabetes……Humanbeingsfightagainstallkindofdiseases■BackgroundChemicaldrugsSurgeryPhysicalTherapyTransplantation therapyIm
2、munotherapyRegenerative medicine……DifferenttreatmentstothediseasesGene therapy■BackgroundDefinition:Genetherapy istheuseof DNA asa drug totreatdiseasebydeliveringtherapeuticDNAintoapatient'scells.Gene therapy※UsingDNAtoexpressfunctionalprotein※UsingsiRNAorshRNAtoattenuateabnormalge
3、neexpressioninmRNAlevel※Genomeeditingtocorrectmutantgenesequence:ZFN,TALEN,CRISPR-Cas9…■BackgroundApplications:Genetherapycanbeappliedinmanydiseases,suchascancer,viraldisease,geneticdisease,etal.Andmostlyappliabletothesingle-gene disorders.Gene therapy※Thefirstapprovedgenetherapyca
4、seintheUnitedStatestookplaceon14September1990.※Therearemorethan2000clinicaltrialshavebeinglaunchedinthepastsevenyears.■BackgroundTechnical problem:BarrierstothetargetDNA/RNAisunstableinthebloodstream,canbeimmunogenicanddoesnotreadilycrossmembranestoentercells.Nuclease,renalfiltrati
5、onPoorselectivityandinefficiencyofenrichmentinthetargetcellortissue.■BackgroundDifferentdeliverytoolsVirusVector:Retrovirus,Lentivirus,Adenovirus,Adeno-associatedvirus(AAV),HerpesSimplexVirus(HSV-1),etal.Non-virusmethod:CyclodextrinPolymers,Lipids,Peptide,Antibodies,Aptamers,andsma
6、llmolecules■BackgroundVirusbasedtransductionWidelystudiedRNAvirusWidehostrangeInfectdividingcellandjustinfectonceIntegrateintohostgenomeLowImmunogenicLongexpressionperiodInsertionmutationbyrandomintegrationandoncogenicityCan’tinfectnon-dividingcellandlowvirustiterRetrovirus:■Backgr
7、oundVirusbasedtransductionRNAvirusderivedfromHIVCaninfectnon-dividingcellLowImmunogenicLongexpressionperiodInsertionmutationbyrandomintegrationandoncogenicityLowvirustiterLentivirus:■BackgroundVirusbasedtransductionDNAvirusHighvirustiter(upto1014VP/ml)andhighinfectionefficiencyWide
8、hostrangeandlargetransgene
