mdx mouse肌营养不良实验鼠课件

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1、Thetreatmentofmusculardystrophy(1)Thetreatmentofpharmaceuticals(2)Genetherapy(3)Celltransplantationtherapy[MesenchymalStemCellTransplanttoTreatDuchenneMuscularDystrophy]Themdxmouse——肌营养不良实验鼠Thesedystrophicmicehaveapointmutationthatexchangedthebasecytosine(C)inposition3,185inexon23againstathy

2、mine(T).ThisconvertedtheCAAcodon,whichnormallycodesfortheaminoacidglutamine,tothetripletTAA(inthemRNAitbecomeUAA)whichisaprematurestopcodon.它是一种点突变实验鼠，基因突变发生在肌营养不良蛋白23号外显子的第3185号核苷酸上，将本该编码为谷氨酸盐氨基酸的CAA码，改变成了TAA码，这是一个停止合成蛋白质的密码，造成了点突变，使得翻译蛋白质的过程提前停止.ThetreatmentofpharmaceuticalsStudiesonmdxmic

3、ehaveturneduponedrug-anantibioticcalledgentamicin(正大霉素)thatcausestheanimalstosynthesizedystrophinandprotectsthemfrommuscledamage.GentamicincureinmdxmiceGentamicinisanantibioticthatcausestheRNAtranslationmechanismintheribosomestoignoresuchaprema-turestopcodon,i.e.toreadthroughitandproduceadys

4、trophinmolecule.Gentamicininjectionsintomdxmiceledtotheappearanceofnewdystrophininupto20%ofthemusclefibers.Andmicewiththisamountofdystrophinhadameliorated改善的clinicalsymptoms.GentamicincureinhumenAbout5%to10%ofDuchenneboyshaveapointmutationintheirdystrophingenewhichchangedanaminoacidcodewordi

5、ntooneofthethreestopcodons.InthemRNA,thesecodonsbecomeUGA,UAG,andUAAandcausetheproteinsynthesistoshutdownprematurely,beforethenewprotein,inthiscasedystrophin,isready.So,wecanusethesamemethodtocurethemusculardystrophyinhumanbeing.OtherdiscoveryaboutthisdrugTheresearchersanalyzedanddiscoveredt

6、hatgentamicinexistsin5varietieswithdifferentstructures.Althoughtheyallhadthesameantibioticactivity,theirreadthroughefficiencywasquitedistinctandtheirtoxicityalso.Thismeansthatgentamicinpreparationswouldhavetobecarefullyselectedbeforetheyareusedforclinicaltrials.ThedisadvantagesofthisdrugAlth

7、oughGentamicinhastheadvantageofbeingawellknowndrugwhosealternativeuseasapossibletherapyforDuchennemusculardystrophy.Asread-throughdoesnotoccuratthegenelevelbutduringproteinsynthesisintheribosomes,treatmentwillhavetoberepeatedperiodically.However,ge