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    • 6. 发明专利
    • Self-inactivating retrovirus vector
    • 自我灭绝逆转录病毒载体
    • JP2007054069A
    • 2007-03-08
    • JP2006228267
    • 2006-08-24
    • Medizinische Hochschule Hannoverメディツィニシェ・ホフシューレ・ハノーバー
    • BAUM CHRISTOPHERSCHAMBACH AXELBOHNE JENS
    • C12N15/09A61K35/76A61K48/00C12N7/00
    • C12N15/86A61K48/00C12N2740/13022C12N2740/13043
    • PROBLEM TO BE SOLVED: To provide a retrovirus vector, especially a self-inactivating (SIN) gammaretroviral or lentiviral vector suitable for producing viral particles of high titers and usable for efficient gene transfer into mammal cells, organs or organisms, e.g. for gene therapy. SOLUTION: More specifically, the invention provides a modified 5'-promoter element in the U3-region of the 5'-LTR of the vector plasmid and a modified 3'-SIN element in the U3-region of the 3'-LTR of the vector plasmid suitable for being comprised in retroviral vectors. Both of the modified 5'-promoter element and the modified 3'-SIN element are preferably contained in retroviral vectors in combination with one another, and the specific advantage of both elements is to increase both the titer of viral particles as well as to increase the expression of a transgene in recipient cells. The transgene is arranged between the modified LTRs of the invention. COPYRIGHT: (C)2007,JPO&INPIT
    • 要解决的问题:提供逆转录病毒载体,特别是适合于产生高滴度的病毒颗粒并可用于将有效基因转移到哺乳动物细胞,器官或生物体中的自灭活(SIN)瘤胃病毒或慢病毒载体。 用于基因治疗。 解决方案:更具体地,本发明提供载体质粒的5'-LTR的U3区中的修饰的5'-启动子元件和3'端的U3区中的修饰的3'-SIN元件, 适合包含在逆转录病毒载体中的载体质粒的-LTR。 修饰的5'-启动子元件和修饰的3'-SIN元件优选彼此组合地包含在逆转录病毒载体中,并且两个元件的具体优点是增加病毒颗粒的滴度以及增加 受体细胞中转基因的表达。 转基因被布置在本发明的修饰的LTR之间。 版权所有(C)2007,JPO&INPIT