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1. 发明申请

WO2011087855A1 METHOD FOR EVALUATING INHIBITORY POLYNUCLEOTIDE EFFICIENCY AND EFFICACY 审中-公开
标题翻译：评估抑制性多核苷酸效率和效率的方法
公开(公告)号：WO2011087855A1
公开(公告)日：2011-07-21
申请号：PCT/US2010/061880
申请日：2010-12-22
申请人： COLUMBIA UNIVERSITY , COHEN, Ira S. , WHITE, Thomas W. , ROBINSON, Richard B. , BRINK, Peter R. , MATHIAS, Richard T. , ROSEN, Michael R. , THE RESEARCH FOUNDATION OF STATE UNIVERSITY OF NEW YORK
发明人： COHEN, Ira S. , WHITE, Thomas W. , ROBINSON, Richard B. , BRINK, Peter R. , MATHIAS, Richard T. , ROSEN, Michael R.
IPC分类号： C12Q1/68
CPC分类号： G01N33/5088 , C12Q1/6897 , C12Q2525/207
摘要： The present invention provides a method for testing the efficiency of delivering an inhibitory polynucleotide to a target cell or tissue. The invention also provides a method for testing efficiency of delivering and efficacy for an effect on tumor size of an inhibitory polynucleotide against a target gene.
摘要翻译：本发明提供了一种测试将抑制性多核苷酸递送至靶细胞或组织的效率的方法。本发明还提供了一种用于检测递送效率的方法以及对靶基因的抑制性多核苷酸对肿瘤大小的影响的效力。

2. 发明申请

WO2013151735A1 METHOD TO MITIGATE LONG-QT ARRHYTHMIAS 审中-公开
标题翻译：减轻长颈椎病的方法
公开(公告)号：WO2013151735A1
公开(公告)日：2013-10-10
申请号：PCT/US2013/031772
申请日：2013-03-14
申请人： THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK , THE RESEARCH FOUNDATION OF STATE UNIVERSITY OF NEW YORK
发明人： COHEN, Ira , ROSEN, Michael R.
IPC分类号： C40B30/06 , G01R27/08 , C12N5/077
CPC分类号： G01N33/5061 , G01N2800/326
摘要： Screening for compounds capable of mitigating LQTS includes incubating cardiomyocytes, providing a test population, and contacting a candidate compound. An outward potassium ion current is measured in response to a depolarizing voltage. If the outward potassium current is increased relative to an outward potassium current in a control population, then it is determined that the candidate compound is capable of treating LQTS. In another embodiment, a method includes contacting an agent that prolongs an action potential to a first test population. After contacting the candidate compound, an action potential is measured. If a duration of the action potential is reduced towards a control duration, then the candidate compound is capable of mitigating LQTS. Some embodiments include determining that the action potential in a second test population with the candidate but not the agent is not different from action potential of a control population.
摘要翻译：筛选能够减轻LQTS的化合物包括孵育心肌细胞，提供测试群体，并与候选化合物接触。响应于去极化电压测量向外的钾离子电流。如果向外的钾电流相对于对照群体中的向外钾电流增加，则确定候选化合物能够处理LQTS。在另一个实施方案中，一种方法包括使延长动作电位的试剂与第一测试群体接触。接触候选化合物后，测定动作电位。如果动作电位的持续时间朝向控制持续时间减小，则候选化合物能够减轻LQTS。一些实施例包括确定第二测试群体中具有候选但不是代理的动作潜力与控制群体的动作潜力没有不同。

3. 发明申请

WO2015109034A1 BIOLOGICAL PACEMAKERS INCORPORATING HCN2 AND SKM1 GENES 审中-公开
标题翻译：包含HCN2和SKM1基因的生物制剂
公开(公告)号：WO2015109034A1
公开(公告)日：2015-07-23
申请号：PCT/US2015/011486
申请日：2015-01-14
申请人： THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK
发明人： ROSEN, Michael R. , TAN, Hanno L. , BOINK, Gerard J. , ROBINSON, Richard B. , COHEN, Ira S. , BRINK, Peter R. , DANILO, JR., Peter
IPC分类号： A61N1/00 , C12N5/00 , A01N63/00
CPC分类号： C07K14/705 , A61K38/177 , A61K48/00 , A61K48/0058 , A61N1/3628 , A61N1/365 , C12N5/0662 , C12N2510/00
摘要： It is demonstrated that hyperpolarization-activated cyclic nucleotide-gated (HCN)-based biological pacing, especially that achieved by transduction of the HCN2 gene into cardiac cells in vivo, was significantly improved by co-transduction of the skeletal muscle sodium channel 1 (SkMI) gene. Expression of both genes hyperpolarized the action potential (AP) threshold. When viral biological pacemaker constructs carrying genes for HCN2 and SkMI were injected into the heart of dogs in vivo, the pacemaker function was facilitated by the slow depolarizing HCN2 current and the hyperpolarized AP threshold generated by SkMI. This dual gene therapy provided both highly efficient pacing and a brisk autonomic response that is superior to those of previously developed gene- or cell-based approaches.
摘要翻译：证明了超极化激活的基于环核苷酸门控（HCN）的生物起搏，特别是通过在体内将HCN2基因转导进入心脏细胞而获得的生物起搏，通过骨骼肌钠通道1（SkMI）的共转导而显着改善）基因。两种基因的表达超极化动作电位（AP）阈值。携带HCN2和SkMI基因的病毒生物起搏器构建体内注射到狗的心脏中，通过慢性去极化HCN2电流和SkMI产生的超极化AP阈值促进起搏器功能。这种双重基因治疗提供了高效起搏和轻微的自主反应，优于以前开发的基于基因或细胞的方法。

4. 发明申请

WO2017165641A1 CANCER TREATMENT BASED ON DELIVERY OF OLIGOES VIA GAP JUNCTIONS FROM HUMAN MESENCHYMAL STEM CELLS (hMSC) 审中-公开
标题翻译：基于人类间充质干细胞（hMSC）间隙连接输送低聚糖的癌症治疗
公开(公告)号：WO2017165641A1
公开(公告)日：2017-09-28
申请号：PCT/US2017/023803
申请日：2017-03-23
申请人： THE TRUSTEES OF COLUMBIA UNIVERSITY IN THE CITY OF NEW YORK , THE RESEARCH FOUNDATION for THE STATE UNIVERSITY OF NEW YORK
发明人： COHEN, Ira S. , BRINK, Peter R. , ROSEN, Michael R.
IPC分类号： A01N43/04 , A61N1/362 , A61P35/00 , C07H21/00 , C07H21/04 , C12P19/34
摘要： A method of treating cancer in vivo includes introducing in vitro into human mesenchymal stem cells (hMSCs) at least one type of inhibitory oligonucleotide, and contacting a tumor tissue of syncytial cancer cells with the hMSCs in vivo under conditions permitting a hMSC to form a gap junction channel with a first syncytial cancer cell of the tumor tissue. As a result, the at least one type of inhibitory oligonucleotide is delivered into the first syncytial cancer cell by traversing the gap junction channel and the at least one type of inhibitory oligonucleotide is delivered into a second syncytial cancer cell of the tumor tissue by traversing a gap junction channel between the first syncytial cancer cell and the second syncytial cancer cell.
摘要翻译：体内治疗癌症的方法包括体外向人间充质干细胞（hMSC）中引入至少一种类型的抑制性寡核苷酸，并使合胞体癌细胞的肿瘤组织与hMSC在体内接触条件允许hMSC与肿瘤组织的第一合胞癌细胞形成间隙连接通道。结果，通过穿过间隙连接通道将至少一种抑制性寡核苷酸递送到第一合胞体癌细胞中，并且通过穿过间隙连接通道将至少一种类型的抑制性寡核苷酸递送到肿瘤组织的第二合胞体癌细胞中第一合胞癌细胞和第二合胞癌细胞之间的间隙连接通道。

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