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    PHARMACOLOGICALLY INDUCED TRANSGENE ABLATION SYSTEM
    2.
    发明申请
    PHARMACOLOGICALLY INDUCED TRANSGENE ABLATION SYSTEM 审中-公开
    药理学诱导转基因系统

    公开(公告)号：WO2011126808A2

    公开(公告)日：2011-10-13

    申请号：PCT/US2011030213

    申请日：2011-03-28

    申请人： UNIV PENNSYLVANIA ,  WILSON JAMES M ,  CHEN SHU-JEN ,  TRETIAKOVA ANNA P

    发明人： WILSON JAMES M ,  CHEN SHU-JEN ,  TRETIAKOVA ANNA P

    IPC分类号： C12N15/86 ,  A61K48/00 ,  C12N15/864

    CPC分类号： C12N15/86 ,  A61K48/0066 ,  C07K2319/715 ,  C07K2319/80 ,  C12N9/22 ,  C12N2750/14143 ,  C12N2800/30 ,  C12N2800/80 ,  C12N2830/002 ,  C12N2830/003 ,  C12N2830/005 ,  C12N2830/006 ,  C12N2830/20 ,  C12N2840/203

    摘要： The present invention relates to gene therapy systems designed for the delivery of a therapeutic product to a subject using replication-defective virus composition(s) engineered with a built-in safety mechanism for ablating the therapeutic gene product, either permanently or temporarily, in response to a pharmacological agent - preferably an oral formulation, e.g., a pill. The invention is based, in part, on the applicants' development of an integrated approach, referred to herein as "PITA" (Pharmacologically Induced Transgene Ablation), for ablating a transgene or negatively regulating transgene expression. In this approach, replication-deficient viruses are used to deliver a transgene encoding a therapeutic product (an RNA or a protein) so that it is expressed in the subject, but can be reversibly or irreversibly turned off by administering the pharmacological agent; e.g., by administration of a small molecule that induces expression of an ablator specific for the transgene or its RNA transcript.

    摘要翻译： 本发明涉及基因治疗系统，其被设计用于使用使用内置安全机制工程改造的复制缺陷型病毒组合物将治疗产品递送给受试者，所述安全机制永久地或临时地用于消融治疗性基因产物 药物剂 - 优选口服制剂，例如丸剂。 本发明部分地基于申请人开发一种综合方法，在本文中称为“PITA”（药理诱导的转基因消融），用于消融转基因或负调控转基因表达。 在这种方法中，使用复制缺陷病毒递送编码治疗产物（RNA或蛋白质）的转基因，使其在受试者中表达，但是可以通过施用药理学试剂来逆转或不可逆地关闭; 例如通过施用诱导特异于转基因或其RNA转录物的消融体表达的小分子。

    PHARMACOLOGICALLY INDUCED TRANSGENE ABLATION SYSTEM
    4.
    发明申请
    PHARMACOLOGICALLY INDUCED TRANSGENE ABLATION SYSTEM 审中-公开
    药理学诱导的转基因消融系统

    公开(公告)号：WO2011126808A3

    公开(公告)日：2012-06-28

    申请号：PCT/US2011030213

    申请日：2011-03-28

    申请人： UNIV PENNSYLVANIA ,  WILSON JAMES M ,  CHEN SHU-JEN ,  TRETIAKOVA ANNA P

    发明人： WILSON JAMES M ,  CHEN SHU-JEN ,  TRETIAKOVA ANNA P

    IPC分类号： C12N15/86 ,  A61K48/00 ,  C12N15/864

    CPC分类号： C12N15/86 ,  A61K48/0066 ,  C07K2319/715 ,  C07K2319/80 ,  C12N9/22 ,  C12N2750/14143 ,  C12N2800/30 ,  C12N2800/80 ,  C12N2830/002 ,  C12N2830/003 ,  C12N2830/005 ,  C12N2830/006 ,  C12N2830/20 ,  C12N2840/203

    摘要： The present invention relates to gene therapy systems designed for the delivery of a therapeutic product to a subject using replication-defective virus composition(s) engineered with a built-in safety mechanism for ablating the therapeutic gene product, either permanently or temporarily, in response to a pharmacological agent - preferably an oral formulation, e.g., a pill. The invention is based, in part, on the applicants' development of an integrated approach, referred to herein as "PITA" (Pharmacologically Induced Transgene Ablation), for ablating a transgene or negatively regulating transgene expression. In this approach, replication-deficient viruses are used to deliver a transgene encoding a therapeutic product (an RNA or a protein) so that it is expressed in the subject, but can be reversibly or irreversibly turned off by administering the pharmacological agent; e.g., by administration of a small molecule that induces expression of an ablator specific for the transgene or its RNA transcript.

    摘要翻译： 本发明涉及基因治疗系统，其被设计用于使用复制缺陷型病毒组合物将治疗产品递送至受试者，所述复制缺陷型病毒组合物用内在的安全机制工程化以永久地或暂时地消融治疗性基因产物， 药理学试剂 - 优选口服制剂，例如丸剂。 本发明部分基于申请人开发的综合方法，在此被称为“PITA”（药理学诱导的转基因消融），用于消融转基因或负调控转基因表达。 在该方法中，使用复制缺陷型病毒来递送编码治疗产物（RNA或蛋白质）的转基因，以使其在受试者中表达，但可以通过施用药理学剂可逆地或不可逆地关闭; 例如通过施用诱导特定于转基因或其RNA转录物的消融因子表达的小分子。