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    • 2. 发明申请
    • IN VIVO GENE SENSORS
    • VIVO基因传感器
    • WO2009137136A3
    • 2013-03-21
    • PCT/US2009034296
    • 2009-02-17
    • UNIV BOSTONMASSACHUSETTS INST TECHNOLOGYCOLLINS JAMES JLU TIMOTHY KUAN-TA
    • COLLINS JAMES JLU TIMOTHY KUAN-TA
    • A61K48/00C12N15/10C12N15/11C12N15/62C12N15/63
    • C12N15/1086C12N15/111C12N15/63C12N2310/111C12N2320/50
    • Described are methods and compositions for the detection of target genes. The inventors have developed a synthetic nucleic acid sensor-effector gene circuit. In cells without a target gene, the circuit suppresses e.g., effector production, but in the presence of the target gene the suppression is subject to competition, such that the synthetic sensor is de-repressed and permits expression of the effector gene. The methods and compositions described further permit the selective expression of an effector gene in those cells expressing the target gene. In this manner, cells expressing a target gene can be selectively targeted for treatment or elimination. In certain aspects, the methods and compositions described permit the selective expression of an agent such as a therapeutic gene product, in a specifically targeted population of cells in an organism.
    • 描述了用于检测靶基因的方法和组合物。 本发明人开发了合成核酸传感器 - 效应子基因电路。 在没有靶基因的细胞中,电路抑制例如效应子产生,但是在靶基因的存在下,抑制受到竞争,使得合成传感器被去抑制并且允许效应基因的表达。 所描述的方法和组合物进一步允许在表达靶基因的那些细胞中选择性表达效应基因。 以这种方式,表达靶基因的细胞可以选择性地靶向用于治疗或消除。 在某些方面,所描述的方法和组合允许在生物体中的特定靶细胞群中选择性地表达诸如治疗性基因产物的试剂。
    • 4. 发明申请
    • IN VIVO GENE SENSORS
    • 体内基因传感器
    • WO2009137136A9
    • 2009-12-30
    • PCT/US2009034296
    • 2009-02-17
    • UNIV BOSTONMASSACHUSETTS INST TECHNOLOGYCOLLINS JAMES JLU TIMOTHY KUAN-TA
    • COLLINS JAMES JLU TIMOTHY KUAN-TA
    • C12Q1/68
    • C12N15/1086C12N15/111C12N15/63C12N2310/111C12N2320/50
    • Described are methods and compositions for the detection of target genes. The inventors have developed a synthetic nucleic acid sensor-effector gene circuit. In cells without a target gene, the circuit suppresses e.g., effector production, but in the presence of the target gene the suppression is subject to competition, such that the synthetic sensor is de-repressed and permits expression of the effector gene. The methods and compositions described further permit the selective expression of an effector gene in those cells expressing the target gene. In this manner, cells expressing a target gene can be selectively targeted for treatment or elimination. In certain aspects, the methods and compositions described permit the selective expression of an agent such as a therapeutic gene product, in a specifically targeted population of cells in an organism.
    • 描述了用于检测靶基因的方法和组合物。 本发明人开发了合成的核酸传感器 - 效应基因电路。 在没有靶基因的细胞中,电路抑制例如效应物产生,但是在靶基因存在下抑制受到竞争,使得合成传感器解除抑制并允许效应子基因表达。 所描述的方法和组合物还允许在表达靶基因的那些细胞中选择性表达效应基因。 以这种方式,表达靶基因的细胞可以选择性地靶向治疗或消除。 在某些方面,所述的方法和组合物允许在生物体中特定靶向的细胞群中选择性表达诸如治疗性基因产物的试剂。