心血管病的基因治疗.pptxVIP

心血管病的基因治疗3 GREAT PROJECTSAPOLLO PROJECTMANHATTAN PROJECTHUMAN GENOME PROJECTGENEA gene is part of a DNA. Genes carry instructions that allow the cells to produce specific proteins. GENEGENE THERAPYGene therapy is an experimental medical intervention that involves modifying the genetic material of living cells to fight disease.GENE GENE GENE 基因治疗的方法目标基因载体转导方法目标基因正常基因代替缺陷基因：LDLR基因用于FH阻遏不利基因的表达：反义核酸阻遏c-myb修复异常基因增加有利基因的表达：VEGF多基因联合治疗：NOS, FGF, t-PA, etc载 体非病毒载体真核表达质粒脂质体共扼载体病毒载体腺病毒腺相关病毒逆转录病毒（MLV、HIV）单纯疱疹病毒痘病毒载 体载 体载 体载 体可调控载体Tet-off/Tet-on系统组织特异性启动子胰岛素启动子肌浆球蛋白启动子胶原结合域-动脉内膜损伤局部