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    • 7. 发明授权
    • Intramuscular delivery of recombinant AAV
    • 肌内递送重组AAV
    • US06506379B1
    • 2003-01-14
    • US09481620
    • 2000-01-12
    • Timothy P. ClacksonMichael GilmanDennis Holt
    • Timothy P. ClacksonMichael GilmanDennis Holt
    • A61K4800
    • C12N9/1205A61K48/00C07K14/715C07K2319/00C12N15/1055C12N15/85C12N2830/002C12N2840/203
    • This invention concerns new configurations for biological switches and provides new methods and materials useful for regulating biological events in animal cells. The invention involves recombinant DNA constructs comprising DNA sequences derived from sequences encoding the proteins FRAP, Tor1, Tor2 and other proteins capable of binding to FKBP:rapamycin, other recombinant DNA constructs comprising DNA sequences encoding part or all of an FKBP protein, the proteins encoded by those constructs, cells (especially animal cells) transformed with one or more of the constructs, small molecules (multivalent multimerizing agents) which bind to and are capable of inducing multimerization of the chimeric proteins, and methods for preparing and using the foregoing, including methods involving the intramuscular delivery of such recombinant DNA constructs in AAV virus particles.
    • 本发明涉及用于生物开关的新配置,并且提供了用于调节动物细胞中的生物学事件的新方法和材料。 本发明涉及包含衍生自编码蛋白质FRAP,Tor1,Tor2和能够结合FKBP:雷帕霉素的其他蛋白质的序列的DNA序列的重组DNA构建体,包含编码部分或全部FKBP蛋白的DNA序列的其他重组DNA构建体,编码 通过这些构建体,用一种或多种构建体转化的细胞(特别是动物细胞),结合并能够诱导嵌合蛋白多聚化的小分子(多价多聚化剂),以及制备和使用前述物质的方法,包括 涉及在AAV病毒颗粒中肌内递送这些重组DNA构建体的方法。