7gene therapy专业英语-2(1).pptVIP免费

Genetherapyistheinsertionofgenesintoanindividual'scellsandtissuestotreatadisease,suchasahereditarydiseaseinwhichadeleteriousmutantalleleisreplacedwithafunctionalone.Althoughthetechnologyisstillinitsinfancy,ithasbeenusedwithsomesuccess.Antisensetherapyisnotstrictlyaformofgenetherapy,butisagenetically-mediatedtherapyandisoftenconsideredtogetherwithothermethods.TypesofgenetherapyGermlinegenetherapyInthecaseofgermlinegenetherapy,germcells,i.e.,spermoreggs,aremodifiedbytheintroductionoffunctionalgenes,whichareordinarilyintegratedintotheirgenomes.Therefore,thechangeduetotherapywouldbeheritableandwouldbepassedontolatergenerations.SomaticgenetherapyInthecaseofsomaticgenetherapy,therapeuticgenesaretransferredintothesomaticcellsofapatient.Anymodificationsandeffectswillberestrictedtotheindividualpatientonly,andwillnotbeinheritedbythepatient'soffspring.Thereareavarietyofdifferentmethodstoreplaceorrepairthegenestargetedingenetherapy.Anormalgenemaybeinsertedintoanonspecificlocationwithinthegenometoreplaceanonfunctionalgene.Thisapproachismostcommon.Anabnormalgenecouldbeswappedforanormalgenethroughhomologousrecombination.Theabnormalgenecouldberepairedthroughselectivereversemutation,whichreturnsthegenetoitsnormalfunction.Theregulation(thedegreetowhichageneisturnedonoroff)ofaparticulargeneVectorsingenetherapyRetrovirusesAdenovirusesAdeno-associatedvirusesLentivirusGenetherapyusinganAdenovirusvector.Anewgeneisinsertedintoanadenovirusvector,whichisusedtointroducethemodifiedDNAintoahumancell.Ifthetreatmentissuccessful,thenewgenewillmakeafunctionalprotein.2002,99:1902-1908Successfultreatmentofmurine-thalassemiaintermediabytransferofthehuman-globingenePlenarypaperChadMay,StefanoRivella,AmyChadburn,andMichelSadelainFromtheDepartmentofHumanGenetics/Medicine,theImmunologyProgram,andtheGeneTransferandSomaticCellEngineeringLaboratory,MemorialSloan-KetteringCancerCenter,andtheDepartmentofPathology,WeillMedicalCollegeofCornellUniversity,NewYork,NewYork.IntroductionThe-thalassemi...