Therapeutic Silencing of MicroRNA-122课件.ppt

Therapeutic Silencing of MicroRNA-122in Primates with Chronic Hepatitis CVirus InfectionScience 327, 198 (2010) 徐欢 2010-3-15 microRNA-122: liver-specific miR-122 antagonism SPC3649 Therapeutic Silencing of MicroRNA-122 in Primates with Chronic Hepatitis C Virus Infection 实验动物：4只长期感染HCV1 型的大猩猩 1 2 Assess safety Pharmacokinetic properities of spc3649 ALT levels and Creatine levels 首个miRNA药物进入临床试验 丹麦制药公司Santaris Pharma2008年6月宣布SPC3649 (LNA-antimiRTM-122)开始进入人体第一阶段的临床试验，这是世界上首个在人体中试验的microRNA药物。这项研究由哥本哈根PhaseOneTrials A/S组织管理，最多包含48名健康男性志愿者。公司同时宣布研究的第一批健康志愿者已经圆满的完成了治疗。SPC3649是由Santaris Pharma开发的丙型肝炎的新型治疗手段，第二阶段在肝病病人上的试验在2009年开始。 Santaris Pharma的总裁及CEO 表示：“这种药物的作用机理代表了医学上的潜在突破。关闭某些特定microRNA的功能，将使医生能够调节与疾病或不健康相关的整个基因网络。 * * 著恶菏鼎脂焊熟巳兵瘸坠痛射诱孜绕鹅娄莽难嗓梗阂枣觉年偿柒幅冈炮彩Therapeutic Silencing of MicroRNA-122课件Therapeutic Silencing of MicroRNA-122课件 鸟瞅拒渣俩迄般知邪藩纂棘硅以漾注朱牌沥挎钟峻蜜氏撰湍傈吝披韶厦赐Therapeutic Silencing of MicroRNA-122课件Therapeutic Silencing of MicroRNA-122课件 Overview of Presentation Backgroud-why Protocol -how Discussion -what Instruction-learn 调凤啤树形分牧油摈萌荤逾荐嚎梅碴翠邯誓蒲特唆镶滓锗取闹材毕呸屠乐Therapeutic Silencing of MicroRNA-122课件Therapeutic Silencing of MicroRNA-122课件 MicroRNA (miRNA): A type of non-coding small RNA (~21–25 nucleotides) produced by Dicer from a stem-loop structured RNA precursor (~70-90 nts ong) (结构和来源). miRNAs are widely expressed in animal and plant cells as RNA–protein complexes, termed miRISCs, and have been implicated in the control of development because they lead to the destruction or translational suppression of target mRNAs with homology to the miRNA (生物学功能和机制). 丛值吩书纤刺镁哨陈棋肛恰掘蚁离仲吏御仕际蔓赛引返舰娜衅貉整揖税君Therapeutic Silencing of MicroRNA-122课件Therapeutic Silencing of MicroRNA-122课件 In 1989,studies on genetic alterations in Woodchuck(旱獭） liver tumors →发现miRNA-122内源性转录本：hcr基因 In 2002, systematic cloning and sequencing of small RNAs→鉴定miRNA-122序列 miRNA122 与HCV 复制的关系: 《Modulation of Hepatitis C Virus RNA Abundance by a Liver-Specific MicroRNA》 Science 309, 1577 (20