Treatment of neurodegenerative disease through intracranial delivery of siRNA
    11.
    发明授权
    Treatment of neurodegenerative disease through intracranial delivery of siRNA 有权
    通过颅内输送siRNA治疗神经变性疾病

    公开(公告)号:US07829694B2

    公开(公告)日:2010-11-09

    申请号:US10852997

    申请日:2004-05-25

    IPC分类号: C07H21/04 C12N15/00

    摘要: The present invention provides devices, small interfering RNA, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The present invention also provides valuable small interfering RNA vectors, and methods for treating neurodegenerative disorders such as Alzheimer's disease, Parkinson's disease, Huntington's disease, Spinocerebellar Ataxia Type 1, Type 2, Type 3, and/or dentatorubral-pallidoluysian atrophy.

    摘要翻译: 本发明提供了用于治疗神经变性疾病的装置,小干扰RNA和用于治疗神经变性疾病的方法,其包括以下步骤:手术植入导管,使得导管的排出部分邻近脑中的预定输注部位,并且通过排出部分 的导管中的预定剂量的至少一种能够抑制至少一种神经变性蛋白产生的物质。 本发明还提供有价值的小干扰RNA载体,以及用于治疗神经变性疾病如阿尔茨海默氏病,帕金森氏病,亨廷顿病,脊髓小脑性共济失调1型,2型,和3型和/或牙髓 - 苍白球囊性萎缩症的方法。

    Methods and kits for linking polymorphic sequences to expanded repeat mutations
    13.
    发明申请
    Methods and kits for linking polymorphic sequences to expanded repeat mutations 审中-公开
    将多态性序列与扩增的重复突变连接的方法和试剂盒

    公开(公告)号:US20080280843A1

    公开(公告)日:2008-11-13

    申请号:US11439858

    申请日:2006-05-24

    IPC分类号: A61K31/7105 C12Q1/68

    摘要: Methods and kits are provided for determining which single nucleotide polymorphism (“SNP”) variant of an allele of a heterozygous patient is on the same mRNA transcript as a disease-causing mutation that is at a remote region of the gene's mRNA comprising a) an allele-specific reverse transcription reaction using an allele-specific primer, and b) analysis of the resulting cDNA product from the reverse transcription reaction at the region of the mutation to determine the presence or absence of the mutation on this allele-specific cDNA product.

    摘要翻译: 提供了方法和试剂盒,用于确定杂合患者的等位基因的单个核苷酸多态性(“SNP”)变体与位于基因mRNA的偏远区域的同一mRNA转录物在同一mRNA转录物上,其包含a) 使用等位基因特异性引物的等位基因特异性逆转录反应,以及b)在突变区域从逆转录反应分析得到的cDNA产物,以确定该等位基因特异性cDNA产物上是否存在突变。

    INTRATHECAL CATHETER
    14.
    发明申请
    INTRATHECAL CATHETER 审中-公开
    内部导管

    公开(公告)号:US20080140008A1

    公开(公告)日:2008-06-12

    申请号:US11951771

    申请日:2007-12-06

    IPC分类号: A61M5/178 A61M31/00 B65D69/00

    摘要: An intrathecal catheter for delivering fluid to, or withdrawing fluid from, the cerebrospinal fluid compartment of a patient, includes a proximal end and a distal portion. The distal portion has an outer diameter of about 1 mm or less, a bending stiffness of about 0.002 pounds per square inch or less, and a distal end. A delivery region is located between the proximal end and the distal end, and a lumen extends from the proximal end to the delivery region. The catheter may further include a coiled structure region to prevent dislodgement of the catheter from a tissue in which the coiled structure is implanted, for example the cisterna magna. The catheter may also include tines to anchor portions of the catheter, for example to a portion of the spinal canal.

    摘要翻译: 用于将流体输送到患者的脑脊液腔室或从患者的脑脊液腔室排出流体的鞘内导管包括近端和远端部分。 远侧部分具有约1mm或更小的外径,约0.002磅/平方英寸或更小的弯曲刚度和远端。 输送区域位于近端和远端之间,并且内腔从近端延伸到输送区域。 导管还可以包括螺旋结构区域,以防止导管从其中植入线圈结构的组织例如大便池移出。 导管还可以包括用于锚定导管的部分的齿,例如到椎管的一部分。

    MICROSYRINGE FOR PRE-PACKAGED DELIVERY OF PHARMACEUTICALS
    15.
    发明申请
    MICROSYRINGE FOR PRE-PACKAGED DELIVERY OF PHARMACEUTICALS 有权
    用于包装递送药物的微生物

    公开(公告)号:US20080119787A1

    公开(公告)日:2008-05-22

    申请号:US11562282

    申请日:2006-11-21

    IPC分类号: A61M5/00

    摘要: A microsyringe includes a body, a barrier movable within the body, and a flow regulator. The body includes a pressurizable chamber, an inlet fluidly connected to the pressurizable chamber, an outlet fluidly connected to the pressurizable chamber, and a cannula fluidly connected to the outlet. The flow regulator is fluidly connected to the cannula to limit the flow of a pharmaceutical through the cannula to a maximum rate. The movable barrier fluidly separates the inlet from the outlet. A pharmaceutical is disposed in a space between the movable barrier and the outlet. The inlet is designed to mechanically and fluidly couple to a hydraulic fluid delivery system. Hydraulic fluid from the inlet pressurizes the chamber, causing the barrier to move and displace the pharmaceutical out of the chamber and into the cannula. The flow regulator ensures that the flow rate of the pharmaceutical through the cannula is not excessive. The movable barrier may be a piston or a collapsible membrane.

    摘要翻译: 微型注射器包括主体,在主体内可移动的障碍物和流量调节器。 主体包括可加压室,流体连接到可加压室的入口,流体连接到可加压室的出口以及流体连接到出口的套管。 流量调节器流体地连接到套管,以将药物通过套管的流动限制到最大速率。 可动挡板使入口与出口流体分离。 药物设置在可移动屏障和出口之间的空间中。 入口被设计成机械和流体地联接到液压流体输送系统。 来自入口的液压流体对腔室加压,使得屏障移动并将药物移出腔室并进入套管。 流量调节器确保药物通过套管的流速不会过大。 可移动屏障可以是活塞或可折叠膜。

    Prioritized rule based method and apparatus for diagnosis and treatment of arrhythmias
    16.
    发明授权
    Prioritized rule based method and apparatus for diagnosis and treatment of arrhythmias 失效
    用于诊断和治疗心律失常的优先规则方法和装置

    公开(公告)号:US06487443B2

    公开(公告)日:2002-11-26

    申请号:US09819433

    申请日:2001-03-28

    IPC分类号: A61B5046

    摘要: An implantable antiarrhythmia device which detects and classifies arrhythmias of the human heart, and delivers appropriate therapy. The device employs a method of arrhythmia classification based on a set of prioritized rules, each of the rules defining a plurality of criteria based upon characteristics of sensed depolarizations of heart tissue, each rule being met when the criteria associated with the rule are met. Some rules, when met, trigger delivery of antiarrhythmia therapy. Other rules, when met, inhibit delivery of antiarrhythmia therapy. The rules may be met simultaneously, and if so, the highest priority rule governs the behavior of the device.

    摘要翻译: 一种可植入的抗心律失常装置,用于检测和分类人心脏的心律失常,并提供适当的治疗。 该装置采用基于一组优先规则的心律失常分类方法,每个规则基于感测到的心脏组织去极化的特征来定义多个标准,当满足与规则相关联的标准时,满足每个规则。 一些规则,当遇到时,触发了抗心律失常治疗的传递。 遇到其他规则时,抑制抗心律失常治疗。 可以同时满足规则,如果是,则最高优先级规则控制设备的行为。

    Methods and sequences to suppress primate Huntington gene expression
    17.
    发明授权
    Methods and sequences to suppress primate Huntington gene expression 有权
    抑制灵长动物亨廷顿基因表达的方法和序列

    公开(公告)号:US08557975B2

    公开(公告)日:2013-10-15

    申请号:US13601198

    申请日:2012-08-31

    IPC分类号: C07H21/04 A61K48/00

    CPC分类号: C12N15/113 C12N2310/14

    摘要: Disclosed herein are sequences, molecules and methods used to suppress the expression of HD genes encoding for huntingtin protein in primates including Macaca mulatto and Homo sapiens. These sequences, molecules and methods aid in the study of the pathogenesis of HD and can also provide a treatment for this disease.

    摘要翻译: 本文公开的是用于抑制编码灵长类动物中的亨廷顿蛋白的HD基因的表达的序列,分子和方法,包括猕猴(Macaca mulatto)和智人(Homo sapiens)。 这些序列,分子和方法有助于HD的发病机制的研究,并且还可以为该疾病提供治疗。

    Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna
    19.
    发明授权
    Compositions, devices and methods for treatment of huntington's disease through intracranial delivery of sirna 失效
    通过颅内输送治疗亨廷顿舞蹈症的组合物,装置和方法

    公开(公告)号:US07732591B2

    公开(公告)日:2010-06-08

    申请号:US11501147

    申请日:2006-08-08

    IPC分类号: C07H21/04 A61K9/22

    摘要: The present invention provides devices, small interfering RNAs, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The present invention also provides valuable small interfering RNA vectors, systems, and methods for treating Huntington's disease in vivo without impairment of cell endoplasmic reticulum, spontaneous motor activity, or locomotor activity of a patient.

    摘要翻译: 本发明提供了用于治疗神经变性疾病的装置,小干扰RNA和用于治疗神经变性疾病的方法,包括以下步骤:手术植入导管,使得导管的排出部分邻近脑中的预定输注部位,并且通过排出部分 的导管中的预定剂量的至少一种能够抑制至少一种神经变性蛋白产生的物质。 本发明还提供了有价值的小干扰RNA载体,系统和用于在体内治疗亨廷顿病的方法,而不损伤患者的细胞内质网,自发运动活动或运动活性。