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    • 7. 发明授权
    • Tissue-vectors specific replication and gene expression
    • 用于组织特异性复制和基因表达的载体
    • US06638762B1
    • 2003-10-28
    • US08974391
    • 1997-11-19
    • Yung-Nien ChangPaul L. HallenbeckCarl M. HayDavid A. Stewart
    • Yung-Nien ChangPaul L. HallenbeckCarl M. HayDavid A. Stewart
    • C12N1500
    • C12N15/86A61K48/00C12N2710/10343C12N2830/008C12N2830/15C12N2830/30C12N2830/32C12N2830/85C12N2840/20C12N2840/44
    • The invention generally relates to cell-specific expression vectors. It particularly relates to targeted gene therapy using recombinant expression vectors and particularly adenovirus vectors. The invention specifically relates to replication-conditional expression vectors and methods for using them. Such vectors are able to selectively replicate in a target cell or tissue to provide a therapeutic benefit in a tissue from the presence of the vector per se or from one or more heterologous gene products expressed from the vector and distributed throughout the tissue. In such vectors, a gene essential for replication is placed under the control of a heterologous tissue-specific transcriptional regulatory sequence. Thus, replication is conditioned on the presence of a factor(s) that induces transcription or the absence of a factor(s) that inhibits transcription of the gene by means of the transcriptional regulatory sequence with this vector; therefore, a target tissue can be selectively treated.
    • 本发明一般涉及细胞特异性表达载体。 它特别涉及使用重组表达载体,特别是腺病毒载体的靶向基因治疗。 本发明具体涉及复制条件表达载体及其使用方法。 这样的载体能够在靶细胞或组织中选择性复制,以从载体本身的存在或从载体表达的一种或多种异源基因产物提供组织中的治疗益处并且分布在整个组织中。 在这样的载体中,将复制必需的基因置于异源组织特异性转录调控序列的控制之下。 因此,复制受到诱导转录或不存在通过与该载体的转录调控序列抑制基因转录的因子的因素的制约; 因此,可以选择性地处理靶组织。