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1. 发明申请

WO2003016539A1 RETROVIRAL VECTORS FOR GENE TRANSFER INTO NEURONAL CELLS 审中-公开
标题翻译：用于基因转移到神经细胞的转染载体
公开(公告)号：WO2003016539A1
公开(公告)日：2003-02-27
申请号：PCT/US2002/007547
申请日：2002-03-13
申请人： THOMAS JEFFERSON UNIVERSITY
发明人： DORNBURG, Ralph, C. , SCHNELL, Matthias, J. , DIETZSCHOLD, Bernhard
IPC分类号： C12N15/85
CPC分类号： C12N15/86 , C12N2740/13043 , C12N2740/13045 , C12N2810/60
摘要： The current methods of gene therapy do not allow for the efficient transduction of nerve cells, thereby limiting treatment of diseases or disorders involving the nervous system. The present invention is a method of treating a disease or disorder wherein an avian retrovirus (spleen necrosis virus, SNV) is engineered to express a rabies virus glycoprotein that allows for specific targeting of nerve cells. Since SNV is not infectious to human cells the retrovirus of the present invention is safe. Further, incorporation of a glycoprotein gene, specifically the N2C gene, and a therapeutic gene(s) of interest into the retroviral vector allows for the specific and efficient transduction of nerve cells with the gene(s) of interest, thereby treating a disease or disorder involving nerve cells.
摘要翻译：目前的基因治疗方法不能有效地转导神经细胞，从而限制了涉及神经系统的疾病或病症的治疗。本发明是一种治疗疾病或病症的方法，其中将禽类逆转录病毒（脾坏死病毒，SNV）工程化以表达允许神经细胞特异性靶向的狂犬病病毒糖蛋白。由于SNV对人体细胞不感染，所以本发明的逆转录病毒是安全的。此外，将糖蛋白基因，特别是N2C基因和目的基因的治疗基因并入逆转录病毒载体中，允许具有目的基因的神经细胞的特异性和有效转导，从而治疗疾病或涉及神经细胞的病症。

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