公开(公告)号：US20060293262A1

公开(公告)日：2006-12-28

申请号：US10533621

申请日：2003-10-29

申请人： Judy Lieberman ,  Manjunath Narasimhaswamy ,  Erwie Song ,  Sang-Kyung Lee ,  Premlata Shankar

发明人： Judy Lieberman ,  Manjunath Narasimhaswamy ,  Erwie Song ,  Sang-Kyung Lee ,  Premlata Shankar

IPC分类号： A61K48/00

CPC分类号： C12N15/1138 ,  A61K38/00 ,  A61M1/14 ,  A61M1/16 ,  A61M1/1609 ,  A61M1/1617 ,  A61M1/3406 ,  A61M1/3609 ,  A61M1/3612 ,  A61M2202/0498 ,  A61M2205/15 ,  A61M2205/33 ,  A61M2205/3317 ,  A61M2205/3324 ,  A61M2205/50 ,  A61M2205/52 ,  C12N15/1132 ,  C12N2310/111 ,  C12N2310/14 ,  C12N2310/53 ,  Y02A50/465

摘要： The present invention is based, at least in part, on the discovery of compositions and methods for the treatment and prevention of infectious diseases or disorders, e.g., HIV infection, AIDS, and AIDS-related diseases. In particular, the present invention pertains to methods of modulating cellular gene expression or protein activity, e.g., CCR5, gene expression or protein activity and/or gene expression or protein activity of a gene or sequence of an infectious agent, in order to treat or prevent infectious diseases or disorders, HIV infection, AIDS, or an AIDS-related disease or disorder. In one embodiment the combination of an RNA interfering agent targeting a cellular gene in combination with an RNA interfering agent targeting a gene or sequence of an infectious agent results in prolonged prevention of infection by an infectious agent The present invention is based on the identification of novel RNA interference agents, e.g., siRNA molecules, which target cellular genes, e.g., chemokine receptors, e.g., the CCR5 gene, and result in inhibition of target gene expression on target gene expressing cells, thereby inhibiting entry of infectious agents, e,g., HIV infection into target cells, prevention infection, and/or suppressing replication in established infection.

摘要翻译： 本发明至少部分地基于发现用于治疗和预防感染性疾病或病症的组合物和方法，例如HIV感染，AIDS和AIDS相关疾病。 具体地，本发明涉及调节细胞基因表达或蛋白活性的方法，例如CCR5，基因表达或蛋白质活性和/或感染因子的基因或序列的基因表达或蛋白质活性，以便治疗或 预防传染病或疾病，艾滋病毒感染，艾滋病或艾滋病相关疾病或病症。 在一个实施方案中，靶向细胞基因的RNA干扰剂与靶向感染因子的基因或序列的RNA干扰剂的组合导致长期预防感染因子的感染本发明基于鉴定小鼠 RNA干扰剂，例如siRNA分子，其靶向细胞基因，例如趋化因子受体，例如CCR5基因，并导致靶基因表达细胞上的靶基因表达的抑制，从而抑制感染因子的进入，例如， ，HIV感染进入靶细胞，预防感染和/或抑制已建立的感染中的复制。