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1、ProtocolsforRecombinantAdenovirus腺病毒基因工程的實驗方法學范雄林,Ph.D,Prof.Email:xionglinfan@yahoo.com.cnTel:027-83650639I.IntroductionofGeneTransfer基因轉移的簡介Genetransfer(基因轉移)isaninvaluabletoolroutinelyusedinvitroorinanimalmodelstoinvestigatethemolecularmechanismsunderlyingdiversebiolo
2、gicalfunctionsinvivogenetransfer(體內基因轉移):istoinsertageneintoanorganismusingavector(thatwilltransferonlytherequiredgenetothedesiredtargetcells).genetherapy(基因治療):whichaimstousegenesastherapeuticentitiesinhumans(istheultimategoalofgenetransfer).Thegoalistodelivergeneticin
3、formationtoatargetcell,eithertoreplaceadefectivefunction(monogenicdisease),ortointroduceanadditionalfunctiontotreat(asincancer)ortoprevent(asinavaccine)diseaseAimsofGenetransferexvivogenetherapy(體外基因治療):targetcellsarefirstextractedfromthepatient.Thedesiredgeneistheninse
4、rtedintothesecells,andoncethetransferiscompleted,thecellsarereturnedtothepatient.Thistechniquehashadpromisingresults,butisrestrictedtoalimitednumberoftargetcelltypesanddiseases(Aiutietal.,2002;Hacein-Bey-Abinaetal.,2002).invivogenetherapy(體內基因治療):thevectorhastobeabletod
5、elivertheselectedgenedirectlyintothetargetcellswithinthewholeorganism.Theintroductionofthetherapeuticgeneintothetargetcellcanbeachievedintwoways:exvivoorinvivoII.Overviewofgenetherapyclinicaltrialsworldwide基因治療的臨床現狀NumberoftrialsinitiatedperyearSince1990,>1500clinicaltr
6、ialsSomedaypeoplewilllookbackontheerabeforegenetherapyinthesamewaywelookbackontheerabeforeantibioticsandvaccines.Itisnowpossibletothinkabouttreatingawholeseriesofdiseaseswithaone-shottherapythatwouldlastalifetime.Dr.RochelleHirschhorn(ProfessorofMedicine,NewYorkUniversi
7、ty)Ashantiwasthefirstpatienttobetreatedwithgenetherapy.ShereceivedinfusionsofTcellsthathadbeentransducedwithageneforADA(anenzymethatshelacks),resultinginanameliorationofthesymptomsofherseverecombinedimmunodeficiency(SCID).AshantideSilva,a4-year-oldgirlreceived11infusion
8、sfromSeptember1990toAugust1992Exvivogenetransfertobonemarrow-derivedCD34+hematopoieticstemcells(HSCs)ofapatien
