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1.发明授权Treatment of neurodegenerative disease through intracranial delivery of SIRNA 有权通过SIRNA的颅内输送治疗神经退行性疾病公开(公告)号：US08119611B2
公开(公告)日：2012-02-21
申请号：US12549110
申请日：2009-08-27
申请人： William F Kaemmerer
发明人： William F Kaemmerer
IPC分类号： C12N15/11
CPC分类号： A61K9/0085 , A61K31/713 , 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 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。
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公开(公告)号：US07988668B2
公开(公告)日：2011-08-02
申请号：US11562282
申请日：2006-11-21
申请人： William F. Kaemmerer
发明人： William F. Kaemmerer
IPC分类号： A61M37/00
CPC分类号： A61M5/14526 , A61M5/141 , A61M5/16813 , A61M2210/0693
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 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。 可移动屏障可以是活塞或可折叠膜。
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3.发明申请Compositions and Methods for Making Therapies Delivered by Viral Vectors Reversible for Safety and Allele-Specificity 有权由病毒载体产生治疗的组合物和方法可逆的安全性和等位基因特异性公开(公告)号：US20100284990A1
公开(公告)日：2010-11-11
申请号：US12580996
申请日：2009-10-16
CPC分类号： A61K31/70 , A61K48/005 , A61K48/0066 , C12N2799/025 , C12N2800/30
摘要： The present invention is directed to compositions, methods and kits for regulation of gene therapies for Huntington's Disease, including, without limitation, reversible gene therapies and allele-specific therapies.
摘要翻译： 本发明涉及用于调节亨廷顿病基因疗法的组合物，方法和试剂盒，包括但不限于可逆基因治疗和等位基因特异性疗法。
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4.发明申请DEVICES, SYSTEMS AND METHODS FOR IMPROVING MEMORY AND/OR COGNITIVE FUNCTION THROUGH BRAIN DELIVERY OF SIRNA 有权用于通过SIRNA的脑输送来改善记忆和/或认知功能的装置，系统和方法公开(公告)号：US20090060987A1
公开(公告)日：2009-03-05
申请号：US11930939
申请日：2007-10-31
申请人： William F. Kaemmerer
发明人： William F. Kaemmerer
IPC分类号： A61K9/127 , A61K31/7105 , A61M1/00
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.
摘要翻译： 本发明涉及用于通过脑递送小干扰RNA的组合物或含有编码小干扰RNA的DNA的载体来改善记忆和/或认知功能的装置，系统和方法。 可以使用用于将组合物直接递送至脑的装置，系统和方法或使用装置，系统，递送方法和递送小干扰RNA的组合物或含有编码小干扰RNA的DNA的载体 血脑屏障。 本发明还提供了有价值的小干扰RNA载体，以及用于降低海马，大脑皮质或其他脑区域中BACE1水平对于改善受试者的记忆和/或认知功能有益的影响的方法。
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公开(公告)号：US20080140048A1
公开(公告)日：2008-06-12
申请号：US11951755
申请日：2007-12-06
IPC分类号： A61M31/00
CPC分类号： A61M27/006 , A61M25/0102 , A61M25/0108 , A61M2025/0059 , A61M2025/0063 , A61M2025/0293
摘要： Methods for delivering first and second fluid compositions to a target location of a subject include delivering the first composition from reservoir of an implantable infusion pump and delivering the second composition through a catheter access port of the implantable infusion device. The access port and reservoir are fluidly coupled to a catheter having a delivery region implanted in the target region. The methods more fully realize the therapeutic potential of infusion devices having both an access port and a reservoir. Strategic use of different agents that compliment the function of each other delivered via either the access port or the reservoir can result in enhanced therapeutic potential.
摘要翻译： 将第一和第二流体组合物递送到受试者的目标位置的方法包括从可植入输注泵的储存器输送第一组合物并且通过可植入输注装置的导管进入口输送第二组合物。 进入口和储存器流体地联接到具有植入目标区域中的输送区域的导管。 所述方法更充分地实现具有进入口和储存器的输注装置的治疗潜力。 不同代理人的战略使用，通过访问端口或储存库交付的功能，可以提高治疗潜力。
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6.发明申请Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity 有权用于通过病毒载体递送的治疗用于安全性和等位基因特异性的组合物和方法公开(公告)号：US20080124379A1
公开(公告)日：2008-05-29
申请号：US11592812
申请日：2006-11-03
CPC分类号： A61K31/70 , A61K48/005 , A61K48/0066
摘要： The present invention is directed to compositions methods and kits for regulation of gene therapies, including, without limitation, reversible gene therapies and allele-specific therapies.
摘要翻译： 本发明涉及用于调节基因治疗的组合物方法和试剂盒，包括但不限于可逆基因治疗和等位基因特异性疗法。
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公开(公告)号：US08419710B2
公开(公告)日：2013-04-16
申请号：US11951755
申请日：2007-12-06
CPC分类号： A61M27/006 , A61M25/0102 , A61M25/0108 , A61M2025/0059 , A61M2025/0063 , A61M2025/0293
摘要： Methods for delivering first and second fluid compositions to a target location of a subject include delivering the first composition from reservoir of an implantable infusion pump and delivering the second composition through a catheter access port of the implantable infusion device. The access port and reservoir are fluidly coupled to a catheter having a delivery region implanted in the target region. The methods more fully realize the therapeutic potential of infusion devices having both an access port and a reservoir. Strategic use of different agents that compliment the function of each other delivered via either the access port or the reservoir can result in enhanced therapeutic potential.
摘要翻译： 将第一和第二流体组合物递送到受试者的目标位置的方法包括从可植入输注泵的储存器输送第一组合物并且通过可植入输注装置的导管进入口输送第二组合物。 进入口和储存器流体地联接到具有植入目标区域中的输送区域的导管。 所述方法更充分地实现具有进入口和储存器的输注装置的治疗潜力。 不同代理人的战略使用，通过访问端口或储存库交付的功能，可以提高治疗潜力。
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8.发明授权Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity 有权用于通过病毒载体递送的治疗用于安全性和等位基因特异性的组合物和方法公开(公告)号：US08324367B2
公开(公告)日：2012-12-04
申请号：US12580996
申请日：2009-10-16
IPC分类号： C07H21/04
CPC分类号： A61K31/70 , A61K48/005 , A61K48/0066 , C12N2799/025 , C12N2800/30
摘要： The present invention is directed to compositions, methods and kits for regulation of gene therapies for Huntington's Disease, including, without limitation, reversible gene therapies and allele-specific therapies.
摘要翻译： 本发明涉及用于调节亨廷顿病基因疗法的组合物，方法和试剂盒，包括但不限于可逆基因治疗和等位基因特异性疗法。
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9.发明申请公开(公告)号：US20120253261A1
公开(公告)日：2012-10-04
申请号：US13074808
申请日：2011-03-29
IPC分类号： A61M37/00
CPC分类号： A61N5/0622 , A61B2017/00039 , A61M5/14276 , A61N5/0601 , A61N5/062 , A61N2005/0612 , A61N2005/063 , A61N2005/0651
摘要： Cells within a patient are optogenetically modulated to treat various neurological disorders. In one example, a method includes delivering a viral vector including a genetic agent encoding for one or more light-sensitive proteins to a delivery site within a patient. The viral vector includes retrograde and/or anterograde transport properties such that the viral vector is configured to transduce the genetic agent into cells at the delivery site and into cells in a plurality of sites proximal and remote to the delivery site. A bioelectrical signal(s) related to a neurological condition of the patient is sensed, e.g. using an implanted electrode. Optical stimulation is delivered to cells transduced with the genetic agent by the viral vector to treat the neurological condition of the patient.
摘要翻译： 患者内的细胞被光电调制以治疗各种神经障碍。 在一个实例中，方法包括将包含编码一种或多种感光蛋白的遗传剂的病毒载体递送至患者内的递送部位。 病毒载体包括逆行和/或顺行转运性质，使得病毒载体被配置为将遗传剂转导到递送部位的细胞中，并转移到多个位点和远离递送部位的细胞中。 感测与患者的神经学状况有关的生物电信号，例如， 使用植入电极。 光学刺激被递送到用病毒载体用遗传剂转导的细胞以治疗患者的神经病学状况。
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10.发明申请Methods and sequences to suppress primate huntington gene expression in vivo 有权在体内抑制灵长类亨廷顿基因表达的方法和序列公开(公告)号：US20100325746A9
公开(公告)日：2010-12-23
申请号：US11501634
申请日：2006-08-09
IPC分类号： A01K67/027 , C07H21/04 , A61K48/00 , C12N15/86
CPC分类号： C12N15/113 , C12N2310/111 , C12N2310/14 , C12N2750/14143 , C12N2750/14171
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 by reducing HD mRNA without causing death, locomotor impairment or cellular alterations of the Macaca mulatta and Homo sapiens.
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，并且还可以通过降低HD mRNA而不引起猕猴猕猴和智人的死亡，运动损伤或细胞改变来为该疾病提供治疗。
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