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    • 8. 发明申请
    • EFFICIENT METHODS FOR ASSESSING AND VALIDATING CANDIDATE PROTEIN-BASED THERAPEUTIC MOLECULES ENCODED BY NUCLEIC ACID SEQUENCES OF INTEREST
    • 用于评估和验证核苷酸基因治疗分子的有效方法由核酸酸序列编码
    • WO03006669A2
    • 2003-01-23
    • PCT/IL0200549
    • 2002-07-07
    • YISSUM RES DEV COMEDGENICS INCGARFINKEL LEONARD IPEARLMAN ANDREW LMITRANI EDUARDO N
    • GARFINKEL LEONARD IPEARLMAN ANDREW LMITRANI EDUARDO N
    • C12N15/10C12Q
    • C12N15/1079C12N15/1034C12N15/1055
    • A method of determining at least one quantitative or qualitative pharmacological, physiological and/or therapeutic, parameter or effect of a recombinant gene product in vivo, the method comprises (a) obtaining at least one micro-organ explant from a donor subject, the micro-organ explant comprising a population of cells, the micro-organ explant maintaining a microarchitecture of an organ from which it is derived and at the same time having dimensions selected so as to allow diffusion of adequate nutrients and gases to cells in the micro-organ explant and diffusion of cellular waste out of the micro-organ explant so as to minimize cellular toxicity and concomitant death due to insufficient nutrition and accumulation of the waste in the micro-organ explant, at least some cells of the population of cells of the micro-organ explant expressing and secreting at least one recombinant gene product; (b) implanting the at least one micro-organ explant in a recipient subject; and (c) determining the at least one quantitative or qualitative pharmacological, physiological and/or therapeutic, parameter or effect of the recombinant gene product in the recipient subject.
    • 一种确定体内重组基因产物的至少一种定量或定性药理,生理和/或治疗,参数或作用的方法,所述方法包括(a)从供体受试者获得至少一种微器官外植体,所述微观 - 包含细胞群的组织外植体,所述微器官外植体保持其来源的器官的微结构,并且同时具有选择的尺寸,以允许足够的营养物和气体扩散到微器官中的细胞 细胞废弃物从微器官外植体中的外植和扩散,以便最小化细胞毒性和伴随死亡,这是由于营养不良和微器官外植体中的废物积累不足,微生物细胞群体的至少一些细胞 表达和分泌至少一种重组基因产物的外植体; (b)将至少一个微器官外植体植入受体受试者; 和(c)确定重组基因产物在受体受试者中的至少一种定量或定性的药理学,生理学和/或治疗学参数或作用。