公开(公告)号：US08957038B2

公开(公告)日：2015-02-17

申请号：US13383560

申请日：2010-07-15

申请人： Dinah W. Y. Sah ,  Andrei P. Guzaev ,  Matthew H. Adams ,  Pei Ge ,  Muthiah Manoharan ,  Douglas Ulen Gwost ,  Gregory Robert Stewart ,  David Kent Stiles ,  Brian Dale Nelson ,  William Frederick Kaemmerer ,  Don Marshall Gash

发明人： Dinah W. Y. Sah ,  Andrei P. Guzaev ,  Matthew H. Adams ,  Pei Ge ,  Muthiah Manoharan ,  Douglas Ulen Gwost ,  Gregory Robert Stewart ,  David Kent Stiles ,  Brian Dale Nelson ,  William Frederick Kaemmerer ,  Don Marshall Gash

IPC分类号： A61K31/70 ,  C07H21/02 ,  C07H21/04 ,  C12N15/113 ,  C12Q1/68

CPC分类号： A61K51/0491 ,  A61K49/04 ,  A61K49/06 ,  A61M5/007 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/346 ,  C12N2310/3517 ,  C12N2320/10 ,  C12N2320/35 ,  C12N2310/3521

摘要： This invention provides treatment compositions as well as systems and methods of determining and administering an effective amount of treatment for a neurological disorder. The treatment composition can contain a labeled interfering RNA (iRNA) agent capable of decreasing expression of a target RNA associated with the neurological disorder. The methods of the invention include determining an effective amount of a therapeutic composition by introducing a solution containing a tracer into the brain of a mammal. The tracing solution is monitored until a target volume of distribution at steady state distribution is substantially achieved, and the rate of delivery of the therapeutic composition is determined. The therapeutic composition can then be administered at the rate determined by use of the tracing solution.

摘要翻译： 本发明提供治疗组合物以及确定和施用有效量的神经障碍治疗的系统和方法。 治疗组合物可以含有能够降低与神经障碍相关的靶RNA表达的标记的干扰RNA（iRNA）试剂。 本发明的方法包括通过将含有示踪剂的溶液引入哺乳动物的脑中来确定治疗组合物的有效量。 监测追踪溶液，直到基本上达到稳定状态分布的目标体积分布，并确定治疗组合物的递送速率。 然后可以以通过使用追踪溶液确定的速率施用治疗组合物。

公开(公告)号：US20120116360A1

公开(公告)日：2012-05-10

申请号：US13383560

申请日：2010-07-15

申请人： Dinah W. Y. Sah ,  Andrei P. Guzaev ,  Matthew H. Adams ,  Pei Ge ,  Muthiah Manoharan ,  Douglas Ulen Gwost ,  Gregory Robert Stewart ,  David Kent Stiles ,  Brian Dale Nelson ,  William Frederick Kaemmerer ,  Don Marshall Gash

发明人： Dinah W. Y. Sah ,  Andrei P. Guzaev ,  Matthew H. Adams ,  Pei Ge ,  Muthiah Manoharan ,  Douglas Ulen Gwost ,  Gregory Robert Stewart ,  David Kent Stiles ,  Brian Dale Nelson ,  William Frederick Kaemmerer ,  Don Marshall Gash

IPC分类号： A61M5/168 ,  A61K49/00 ,  A61M5/142 ,  A61K31/7088

CPC分类号： A61K51/0491 ,  A61K49/04 ,  A61K49/06 ,  A61M5/007 ,  C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/315 ,  C12N2310/321 ,  C12N2310/346 ,  C12N2310/3517 ,  C12N2320/10 ,  C12N2320/35 ,  C12N2310/3521

摘要： This invention provides treatment compositions as well as systems and methods of determining and administering an effective amount of treatment for a neurological disorder. The treatment composition can contain a labeled interfering RNA (iRNA) agent capable of decreasing expression of a target RNA associated with the neurological disorder. The methods of the invention include determining an effective amount of a therapeutic composition by introducing a solution containing a tracer into the brain of a mammal. The tracing solution is monitored until a target volume of distribution at steady state distribution is substantially achieved, and the rate of delivery of the therapeutic composition is determined. The therapeutic composition can then be administered at the rate determined by use of the tracing solution.

摘要翻译： 本发明提供治疗组合物以及确定和施用有效量的神经障碍治疗的系统和方法。 治疗组合物可以含有能够降低与神经障碍相关的靶RNA表达的标记的干扰RNA（iRNA）试剂。 本发明的方法包括通过将含有示踪剂的溶液引入哺乳动物的脑中来确定治疗组合物的有效量。 监测追踪溶液，直到基本上达到稳定状态分布的目标体积分布，并确定治疗组合物的递送速率。 然后可以以通过使用追踪溶液确定的速率施用治疗组合物。