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SYSTEM FOR THE TRANSFER AND EXPRESSION OF HETEROLOGIC GENES IN THE BONE MARROW IN VIVO

机译:异体LOGO骨基因在体内的表达和转移系统

摘要

The present invention provides a method of delivering immunogenic or therapeutic proteins to bone marrow cells using alphavirus vectors. The alphavirus vectors disclosed herein target specifically to bone marrow tissue, and viral genomes persist in bone marrow for at least three months post-infection. No or very low levels of virus were detected in quadricep, brain, and sera of treated animals. The sequence of a consensus Sindbis cDNA clone, pTR339, and infectious RNA transcripts, infectious virus particles, and pharmaceutical formulations derived therefrom are also disclosed. The sequence of the genomic RNA of the Girdwood S.A. virus, and cDNA clones, infectious RNA transcripts, infectious virus particles, and pharmaceutical formulations derived therefrom are also disclosed.
机译:本发明提供了一种使用α病毒载体将免疫原性或治疗性蛋白质递送至骨髓细胞的方法。本文公开的α病毒载体特异性地靶向骨髓组织,并且病毒基因组在骨髓中持续感染后至少三个月。在接受治疗的动物的四头肌,大脑和血清中未检测到病毒或病毒水平非常低。还公开了共有的Sindbis cDNA克隆,pTR339和感染性RNA转录物,感染性病毒颗粒以及从其衍生的药物制剂的序列。还公开了Girdwood S.A.病毒的基因组RNA的序列,以及cDNA克隆,感染性RNA转录物,感染性病毒颗粒以及由此衍生的药物制剂。

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