血红蛋白病血红蛋白复旦大学精品课程HemoglobindiseaseFudanUniversityppt课件.ppt

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1、19基因治疗GeneTherapy1.Whatisgenetherapy?Genes,whicharecarriedonchromosomes,arethebasicphysicalandfunctionalunitsofheredity.Genesarespecificsequencesofbasesthatencodeinstructionsonhowtomakeproteins.Althoughgenesgetalotofattention,it’stheproteinsthatperformmostlif

2、efunctionsandevenmakeupthemajorityofcellularstructures.Whengenesarealteredsothattheencodedproteinsareunabletocarryouttheirnormalfunctions,geneticdisorderscanresult.Genetherapyisatechniqueforcorrectingdefectivegenesresponsiblefordiseasedevelopment.Researchersm

3、ayuseoneofseveralapproachesforcorrectingfaultygenes:Anormalgenemaybeinsertedintoanonspecificlocationwithinthegenometoreplaceanonfunctionalgene.Thisapproachismostcommon.Anabnormalgenecouldbeswappedforanormalgenethroughhomologousrecombination.Theabnormalgenecou

4、ldberepairedthroughselectivereversemutation,whichreturnsthegenetoitsnormalfunction.Theregulation(thedegreetowhichageneisturnedonoroff)ofaparticulargenecouldbealtered.2.Howdoesgenetherapywork?Inmostgenetherapystudies,a"normal"geneisinsertedintothegenometorepla

5、cean"abnormal,"disease-causinggene.Acarriermoleculecalledavectormustbeusedtodeliverthetherapeuticgenetothepatient'stargetcells.Currently,themostcommonvectorisavirusthathasbeengeneticallyalteredtocarrynormalhumanDNA.Viruseshaveevolvedawayofencapsulatinganddeli

6、veringtheirgenestohumancellsinapathogenicmanner.Scientistshavetriedtotakeadvantageofthiscapabilityandmanipulatethevirusgenometoremovedisease-causinggenesandinserttherapeuticgenes.Targetcellssuchasthepatient'sliverorlungcellsareinfectedwiththeviralvector.Theve

7、ctorthenunloadsitsgeneticmaterialcontainingthetherapeutichumangeneintothetargetcell.Thegenerationofafunctionalproteinproductfromthetherapeuticgenerestoresthetargetcelltoanormalstate.Toreversediseasecausedbygeneticdamage,researchersisolatenormalDNAandpackageit

8、intoavector,amoleculardeliverytruckusuallymadefromadisabledvirus.Doctorstheninfectatargetcell—usuallyfromatissueaffectedbytheillness,suchasliverorlungcells—withthevector.Thevectorunloadsi