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    • 4. 发明申请
    • IMPROVED RETROVIRAL VECTORS, ESPECIALLY SUITABLE FOR GENE THERAPY
    • 改进的退化载体,特别适用于基因治疗
    • WO1996035798A1
    • 1996-11-14
    • PCT/NL1996000195
    • 1996-05-07
    • INTROGENE B.V.VOGELS, RonaldBOESEN, Johannes, Jozephes, BernardusVAN ES, Helmuth, Hendrikus, GerardusVAN BEUSECHEM, Victor, WillemVALERIO, Domenico
    • INTROGENE B.V.
    • C12N15/86
    • C12N15/86A61K48/00C12N7/00C12N2740/13023C12N2740/13043C12N2840/20C12N2840/203
    • The present invention relates to the field of molecular biology, especially recombinant DNA technology, especially concerning retroviral vectors. Retroviral vectors are very suitable vehicles for transferring genetic material of interest into certain cells in so-called gene therapy strategies. However, the retroviral vectors described so far are not ideal. They may give rise to recombination events resulting in helper (pathogenic) virus, they may express viral proteinaceous materials leading to immune responses, etc. These and other drawbacks are overcome by the vectors, cells, kits and methods of the present invention by providing a vector derived from a retrovirus, comprising a sequence responsible for transcriptional control, including an enhancer, which vector further comprises a site for insertion of at least one gene of interest, a packaging signal, said vector having no superfluous retroviral sequences and no open reading frame encoding at least parts of viral proteins, characterized in that the enhancer is an enhancer that is active in undifferentiated cells.
    • 本发明涉及分子生物学领域,特别是涉及逆转录病毒载体的重组DNA技术领域。 逆转录病毒载体是用于将遗传物质转移到所谓基因治疗策略中的某些细胞的非常合适的载体。 然而,迄今描述的逆转录病毒载体并不理想。 它们可能引起导致辅助(致病性)病毒的重组事件,它们可以表达导致免疫应答的病毒蛋白质物质等。这些和其它缺点通过本发明的载体,细胞,试剂盒和方法来克服, 衍生自逆转录病毒的载体,包含负责转录控制的序列,包括增强子,所述载体还包含用于插入至少一种目标基因的位点,包装信号,所述载体不具有多余的逆转录病毒序列,并且没有开放阅读框 编码至少部分病毒蛋白质,其特征在于增强子是在未分化细胞中有活性的增强子。