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《基因治療Gene Therapy.ppt》由會(huì)員上傳分享，免費(fèi)在線閱讀，更多相關(guān)內(nèi)容在行業(yè)資料-天天文庫(kù)。
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
