重组腺病毒理论课件

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1、ProtocolsforRecombinantAdenovirus腺病毒基因工程的实验方法学范雄林,Ph.D,Prof.Email:xionglinfan@yahoo.com.cnTel:027-83650639I.IntroductionofGeneTransfer基因转移的简介Genetransfer(基因转移)isaninvaluabletoolroutinelyusedinvitroorinanimalmodelstoinvestigatethemolecularmechanismsunderlyingdiversebiolo

2、gicalfunctionsinvivogenetransfer(体内基因转移):istoinsertageneintoanorganismusingavector(thatwilltransferonlytherequiredgenetothedesiredtargetcells).genetherapy(基因治疗):whichaimstousegenesastherapeuticentitiesinhumans(istheultimategoalofgenetransfer).Thegoalistodelivergeneticin

3、formationtoatargetcell,eithertoreplaceadefectivefunction(monogenicdisease),ortointroduceanadditionalfunctiontotreat(asincancer)ortoprevent(asinavaccine)diseaseAimsofGenetransferexvivogenetherapy(体外基因治疗):targetcellsarefirstextractedfromthepatient.Thedesiredgeneistheninse

4、rtedintothesecells,andoncethetransferiscompleted,thecellsarereturnedtothepatient.Thistechniquehashadpromisingresults,butisrestrictedtoalimitednumberoftargetcelltypesanddiseases(Aiutietal.,2002;Hacein-Bey-Abinaetal.,2002).invivogenetherapy(体内基因治疗):thevectorhastobeabletod

5、elivertheselectedgenedirectlyintothetargetcellswithinthewholeorganism.Theintroductionofthetherapeuticgeneintothetargetcellcanbeachievedintwoways:exvivoorinvivoII.Overviewofgenetherapyclinicaltrialsworldwide基因治疗的临床现状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