公开(公告)号：EP2239328A3

公开(公告)日：2011-01-05

申请号：EP10162816.2

申请日：2006-08-18

申请人： Alnylam Pharmaceuticals Inc. ,  University of Massachusetts

发明人： Manoharan, Muthiah ,  Aronin, Neil

IPC分类号： C12N15/11

CPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K47/554 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2320/32 ,  C12Q1/6883 ,  C12Q2600/136 ,  C12Q2600/158 ,  C12Q2600/178

摘要： This invention relates to methods and compositions for treating neurological disease, and more particularly to methods of delivering iRNA agents to neural cells for the treatment of neurological diseases.

公开(公告)号：EP2239328A2

公开(公告)日：2010-10-13

申请号：EP10162816.2

申请日：2006-08-18

申请人： Alnylam Pharmaceuticals Inc. ,  University of Massachusetts

发明人： Manoharan, Muthiah ,  Aronin, Neil

IPC分类号： C12N15/11

CPC分类号： C12N15/113 ,  A61K31/7088 ,  A61K47/554 ,  C12N15/111 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/3515 ,  C12N2320/32 ,  C12Q1/6883 ,  C12Q2600/136 ,  C12Q2600/158 ,  C12Q2600/178

摘要： This invention relates to methods and compositions for treating neurological disease, and more particularly to methods of delivering iRNA agents to neural cells for the treatment of neurological diseases.

摘要翻译： 本发明涉及用于治疗神经疾病的方法和组合物，更具体地涉及将iRNA试剂递送至神经细胞用于治疗神经疾病的方法。

公开(公告)号：EP2821085A3

公开(公告)日：2015-04-22

申请号：EP14168602.2

申请日：2004-09-13

申请人： University of Massachusetts

发明人： Aronin, Neil ,  Zamore, Phillip D

IPC分类号： A61K48/00 ,  C07H21/04 ,  C12N15/11

CPC分类号： C07H21/04 ,  A61K48/00 ,  C12N15/113 ,  C12N2310/14

摘要： The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.

公开(公告)号：EP2821085A2

公开(公告)日：2015-01-07

申请号：EP14168602.2

申请日：2004-09-13

申请人： University of Massachusetts

发明人： Aronin, Neil ,  Zamore, Phillip D

IPC分类号： A61K48/00 ,  C07H21/04 ,  C12N15/11

CPC分类号： C07H21/04 ,  A61K48/00 ,  C12N15/113 ,  C12N2310/14

摘要： The present invention relates to the discovery of an effective treatment for a variety of gain-of-function diseases, in particular, Huntington's disease (HD). The present invention utilizes RNA Interference technology (RNAi) against polymorphic regions in the genes encoding various gain-of-function mutant proteins resulting in an effective treatment for the gain-of-function disease.

摘要翻译： 本发明涉及多种功能性增益疾病，特别是亨廷顿病（HD）的有效治疗的发现。 本发明利用RNA干扰技术（RNAi）针对编码各种增益功能突变蛋白质的基因中的多态性区域，导致功能性增益疾病的有效治疗。