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1、GENETHERAPYBySamLaiandMengLeeGeneTherapyIntroduction:Whatisit?GeneTherapy:ACloserLookHowmuchdidyoulearn?!CaseStudy:SCIDModelforGeneTherapyMedicalFrameworkDiscussion/Q&AWhatisit?Genetherapy,whichwasfirstconceivedinthemid-1970s,aimstoinsertworkingcopiesofgenesintocellscontainingdysfun
2、ctionalonestreat,cure,orultimatelypreventdiseasebychangingtheexpressionofaperson'sgenescurrentgenetherapyisprimarilyexperimental,withmosthumanclinicaltrialsonlyintheresearchstagesMostoftenincontextoftreatinglethalanddisablingdiseases,potentialalsofordiseasepreventionHumanGenomeProje
3、ctAgenomeisalltheDNAinanorganism,includingitsgenes.GenescarryinformationformakingalltheproteinsrequiredbyallorganismsVariationsinstructureofperson’sgenescollectivelyhelpsdefineusasindividualsRationaleforGTisbasedonknowledgeofthehumangeneticcodeBeganformallyin1990,setasa13yearproject
4、.HGPGOALSIdentifyaproximately30,000genesDetermine3billionchemicalbasepairsthatmakeuphumanDNAStoreinformationindatabasesImprovetoolsfordataanalysisAddressethical,legalandsocialissuesthatmightarisefromthisHGPcont…Achievethesegoals,researcherslookatgeneticmakeupofotherorganismsThisproj
5、ectisimportantbecausethegovernment'sdedicationtothetransferoftechnologytotheprivatesectorWhichCellsTargeted?somaticgenetherapytherecipient'sgenomeischanged,butthechangeisnotpassedalongtothenextgenerationgermlinegenetherapy,thesexcellsarechangedwiththegoalofpassingthesechangestotheir
6、offspring.But,thisisnotbeingactivelyinvestigated,atleastnothumans,althoughalotofdiscussionisbeingconductedaboutitsvalueanddesirability.SomaticStemCellsSomaticstemcellsareremarkablecellsinthebodyThesedifferentstemcelltypesaretargetsforgenetherapyGeneticallytreatedstemcells,whenreintr
7、oducedintothepatient'sbodyareexpectedtonaturallytravelthroughthebloodstreamtothebonemarrow.GermlineGeneTherapyGenetherapyingermlinecellshasthepotentialtoaffectnotonlytheindividual,butalsotheirchildrenaswell.Anygeneticchangesinthereproductivecellsorchangesmadetotheembryobeforethestag
8、eofdifferentiation,