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公开(公告)号：US20060178328A1
公开(公告)日：2006-08-10
申请号：US11253393
申请日：2005-10-19
申请人： William Kaemmerer
发明人： William Kaemmerer
CPC分类号： C12N15/1137 , A61M31/002 , C12N15/111 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/32
摘要： The present invention relates to devices, systems, and methods for improving memory and/or cognitive function by brain delivery of compositions of small interfering RNA or vectors containing the DNA encoding for small interfering RNA. Such compositions can be administered using devices, systems and methods for direct delivery of the compositions to the brain, or using devices, systems, methods of delivery, and compositions that deliver small interfering RNA or vectors containing the DNA encoding the small interfering RNA across the blood-brain barrier. The present invention also provides valuable small interfering RNA vectors, and methods for reduction of BACE1 levels in the hippocampus, cerebral cortex, or other regions of the brain that have beneficial effects on improving memory and/or cognitive function in a subject.
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2.发明申请System and method for delivering polynucleotides to the central nervous system 审中-公开将多核苷酸递送至中枢神经系统的系统和方法公开(公告)号：US20050048641A1
公开(公告)日：2005-03-03
申请号：US10962732
申请日：2004-10-12
IPC分类号： A61K31/713 , A61M37/00 , C12N15/11 , C12M1/00
CPC分类号： A61M37/00 , A61K31/713
摘要： Methods and apparatuses for delivering RNA polynucleotides to a patient in need thereof are described. Programmable infusion pump systems that include a reservoir housing the RNA polynucleotide are implanted in the patient. The RNA polynucleotide is delivered to a target location in the patient via a catheter in communication with the reservoir. The pump system may include one or more sensors that may control rate or timing of delivery of the RNA polynucloetide based on a detected event. The pump system allows for controlled delivery of RNA polynucleotides for the treatment of diseases, disorders, or conditions.
摘要翻译： 描述了将RNA多核苷酸递送给有需要的患者的方法和装置。 包括容纳RNA多核苷酸的储存器的可编程输液泵系统植入患者体内。 通过与储存器连通的导管将RNA多核苷酸递送至患者的目标位置。 泵系统可以包括可以基于检测到的事件来控制RNA多核苷酸的递送速率或定时的一个或多个传感器。 泵系统允许用于治疗疾病，病症或病症的RNA多核苷酸的受控递送。
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3.发明申请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：US20070167389A1
公开(公告)日：2007-07-19
申请号：US11501147
申请日：2006-08-08
申请人： William Kaemmerer
发明人： William Kaemmerer
CPC分类号： A61K31/713 , A61K9/0085 , A61K48/00 , A61K48/0075 , A61M37/00 , C12N15/111 , C12N15/113 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/32
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。
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4.发明申请公开(公告)号：US20060257912A1
公开(公告)日：2006-11-16
申请号：US11429491
申请日：2006-05-04
申请人： William Kaemmerer , Michael Kaytor
发明人： William Kaemmerer , Michael Kaytor
IPC分类号： A61K48/00 , C12Q1/68 , C07H21/04 , C12P21/06 , C07K14/705
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 mulatta 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： 本文公开了用于抑制编码灵长类动物（包括猕猴属和智人）中的亨廷顿蛋白的HD基因的表达的序列，分子和方法。 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。
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公开(公告)号：US20060079927A1
公开(公告)日：2006-04-13
申请号：US10961964
申请日：2004-10-08
申请人： William Kaemmerer , Kenneth Heruth
发明人： William Kaemmerer , Kenneth Heruth
IPC分类号： A61M29/00
CPC分类号： A61F2/95
摘要： A tip delivery system includes a tip, a tip support structure and a dissolvable joint connecting the tip to the tip support structure. To separate the tip from the tip support structure, a dissolving liquid is delivered to the dissolvable joint. The dissolving liquid reacts, e.g., enzymatically, with the dissolvable joint and dissolves the dissolvable joint.
摘要翻译： 尖端传送系统包括尖端，尖端支撑结构和将尖端连接到尖端支撑结构的可溶解接头。 为了将尖端与尖端支撑结构分开，将溶解液输送到可溶解的接头。 溶解液例如通过酶促与可溶解的接头反应并溶解可溶解的接头。
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公开(公告)号：US20060018882A1
公开(公告)日：2006-01-26
申请号：US11157608
申请日：2005-06-21
CPC分类号： C12N15/86 , A61K48/0033 , A61K48/0075 , A61K48/0083 , C12N15/111 , C12N15/1137 , C12N15/88 , C12N2310/111 , C12N2310/14 , C12N2310/53 , C12N2320/32 , C12N2750/14143
摘要： The present invention provides medical devices and methods for delivering compositions to cells. The compositions include an artificial viral vector, and particularly, an artificial adeno-associated virus vector. Such compositions can be useful for delivering the artificial viral vector across the blood-brain barrier.
摘要翻译： 本发明提供了将组合物递送到细胞的医疗装置和方法。 组合物包括人造病毒载体，特别是人造腺相关病毒载体。 这样的组合物可用于将人造病毒载体递送穿过血脑屏障。
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7.发明申请Methods and systems for treatment of neurological diseases of the central nervous system 审中-公开治疗中枢神经系统神经疾病的方法和系统公开(公告)号：US20050208090A1
公开(公告)日：2005-09-22
申请号：US10803711
申请日：2004-03-18
申请人： John Keimel , William Kaemmerer
发明人： John Keimel , William Kaemmerer
CPC分类号： A61K38/1709
摘要： The present invention is directed to methods and systems for the treatment of inborn genetic errors or other defects that cause deficiencies of active enzymes or proteins within the cells of the central nervous system. Such methods and systems generally comprise an implantable catheter system designed for the chronic delivery of specially formulated proteins to intrathecal, intracerebroventricular, and/or intraparenchymal regions of the central nervous system. The invention has application in the neuropathic aspects of the broad category of lysosomal storage diseases. These genetic based diseases are the result of insufficient enzyme activity to catabolize specific substances, which thereby accumulate in the cellular lysosomes.
摘要翻译： 本发明涉及用于治疗在中枢神经系统的细胞内引起活性酶或蛋白质缺乏的先天遗传错误或其他缺陷的方法和系统。 这样的方法和系统通常包括可植入导管系统，其被设计用于将特殊配制的蛋白质慢性递送至中枢神经系统的鞘内，脑室内和/或实质内区域。 本发明适用于广泛类型的溶酶体贮积病的神经病学方面。 这些基于遗传的疾病是由于酶活性不足而导致特定物质分解代谢的结果，从而在细胞溶酶体中积累。
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8.发明申请公开(公告)号：US20060198825A1
公开(公告)日：2006-09-07
申请号：US11071609
申请日：2005-03-03
CPC分类号： C12N15/113 , C12N2310/111 , C12N2310/14 , C12N2310/53
摘要： The present invention relates to reagents, methods and systems to treat heart failure using small interfering RNA (siRNA) molecules targeted to phospholamban.
摘要翻译： 本发明涉及使用靶向磷胆兰的小干扰RNA（siRNA）分子治疗心力衰竭的试剂，方法和系统。
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9.发明申请Methods of treating cardiac disorders by suppressing the expression of the potassium inwardly-rectifying channel 审中-公开通过抑制钾内向整流通道的表达来治疗心脏疾病的方法公开(公告)号：US20050244377A1
公开(公告)日：2005-11-03
申请号：US11022368
申请日：2004-12-22
申请人： Daniel Sigg , James Coles , Michael Ujhelyi , Rudy Padua , Daisy Cross , William Kaemmerer
发明人： Daniel Sigg , James Coles , Michael Ujhelyi , Rudy Padua , Daisy Cross , William Kaemmerer
IPC分类号： A61K48/00 , C12N15/113 , C12N15/86
CPC分类号： C12N15/1138 , C12N2310/111 , C12N2310/14 , C12N2310/53
摘要： The present invention is directed toward methods for regulating biological pacemaking activity and devices used in such regulation. Such regulation can be accomplished by introducing genetic material to the heart by transfecting heart cells of the atrium or ventricle with an oligonucleotide, small interfering RNA, that silence KCNJ2, and suppress the IK1 current. Suppression (or silencing) of KCNJ2 subsequently induces pacemaker-like activities in previously regular myocytes. This invention provides for methods of targeted delivery using a fluid delivery catheter. Such a catheter allows the targeting of a specific area in the atrium or the ventricle of the heart. Also, combination methods of treating arrhythmia with traditional device-based therapies (e.g., pacemakers and defibrillators) and an oligonucleotide of the subject invention.
摘要翻译： 本发明涉及用于调节生物起搏活性的方法和用于这种调节的装置。 通过用心脏或心室的心脏细胞转染心脏的遗传物质可以通过用沉默KCNJ2的寡核苷酸，小干扰RNA，抑制Iκ1电流来引入遗传物质来实现这种调节。 KCNJ2的抑制（或沉默）随后在以前的正常心肌细胞中诱导起搏器样活性。 本发明提供了使用流体输送导管进行靶向递送的方法。 这样的导管允许靶向心房的心房或心室的特定区域。 此外，用传统的基于装置的疗法（例如起搏器和除颤器）治疗心律失常的组合方法和本发明的寡核苷酸。
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