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1. 发明申请

WO2018144864A1 METHODS FOR MANIPULATING CELL FATE 审中-公开
公开(公告)号：WO2018144864A1
公开(公告)日：2018-08-09
申请号：PCT/US2018/016644
申请日：2018-02-02
申请人： THE MCLEAN HOSPITAL CORPORATION
发明人： KIM, Kwang-Soo
IPC分类号： A61K38/57 , C07K14/435 , C07K14/47 , C12N5/07 , C12N5/071
摘要： Disclosed herein are methods of generating induced pluripotent stem cells. The method involves providing a somatic or non-embryonic cell population, contacting the somatic or non-embryonic cell population with a quantity of at least one reprogramming factor, an agent that downmodulates SIRT2, and/or an agent that upmodulates SIRT1, and culturing the somatic or non-embryonic cells for a period of time sufficient to generate at least one induced pluripotent stem cell. Methods for differentiating a cell by upmodulating SIRT2 and/or downmodulating SIRT1 are also provided herein. Also disclosed are cell lines and pharmaceutical compositions generated by use of the methods.

2. 发明申请

WO2017087282A1 ANTISENSE COMPOUNDS TARGETING LEUCINE-RICH REPEAT KINASE 2 (LRRK2) FOR THE TREATMENT OF PARKINSONS DISEASE 审中-公开
标题翻译：反义化合物靶向富含重复激酶的激酶2（LRRK2）治疗帕金森氏病
公开(公告)号：WO2017087282A1
公开(公告)日：2017-05-26
申请号：PCT/US2016/061664
申请日：2016-11-11
申请人： ROSALIND FRANKLIN UNIVERSITY OF MEDICINE AND SCIENCE , THE MCLEAN HOSPITAL CORPORATION
发明人： HASTINGS, Michelle, L. , ISACSON, Ole , KORECKA-ROET, Joanna, A.
IPC分类号： C12N15/113 , A61K31/712 , A61K31/7125 , C07H21/00 , A61P25/28
CPC分类号： C12N15/1137 , C12N2310/11 , C12N2310/315 , C12N2310/3231 , C12N2310/3233 , C12N2310/346 , C12N2320/33 , C12Y207/11001 , C12N2310/321 , C12N2310/3521 , C12N2310/3525 , C12N2310/322 , C12N2310/3533
摘要： The present disclosure relates generally to compounds comprising oligonucleotides complementary to a Leucine-Rich-Repeat-Kinase (LRRK2) RNA transcript. Certain such compounds are useful for hybridizing to a LRRK2 RNA transcript, including but not limited to a LRRK2 RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the LRRK2 transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Parkinson's disease.
摘要翻译：本公开一般涉及包含与富亮氨酸重复激酶（LRRK2）RNA转录物互补的寡核苷酸的化合物。某些此类化合物可用于与LRRK2RNA转录物杂交，包括但不限于细胞中的LRRK2RNA转录物。在某些实施方案中，这种杂交导致调节LRRK2转录物的剪接。在某些实施方案中，这些化合物用于治疗与帕金森氏病有关的一种或多种症状。

3. 发明申请

WO2016179497A1 GLUCOCEREBROSIDASE GENE THERAPY FOR PARKINSON'S DISEASE 审中-公开
标题翻译：帕金森病的葡萄糖替代酶基因治疗
公开(公告)号：WO2016179497A1
公开(公告)日：2016-11-10
申请号：PCT/US2016/031223
申请日：2016-05-06
申请人： SHIRE HUMAN GENETIC THERAPIES, INC. , THE MCLEAN HOSPITAL CORPORATION
发明人： PARK, Yung Hee , ISACSON, Ole
IPC分类号： C12N15/864 , A61K38/47 , A61K48/00 , A61K35/76 , A61P25/16 , C12N9/24
CPC分类号： A61K48/0058 , A01K2227/105 , A01K2267/0356 , A61K9/0085 , A61K35/76 , A61K38/47 , A61P25/16 , C12N9/2402 , C12N9/2405 , C12N15/86 , C12N2750/14143 , C12N2750/14171 , C12Y302/01045
摘要： The present invention is directed, in part, to the treatment of a subject having a neurodegenerative disorder, such as Parkinson's disease (PD), by providing glucocerebrosidase enzyme. The enzyme may be provided, e.g., through gene therapy or by administration of a glucocerebrosidase protein. Accordingly, the present invention encompasses glucocerebrosidase nucleic acids or proteins for use in the treatment of PD or other neurodegenerative disorders.
摘要翻译：本发明部分地涉及通过提供葡糖脑苷脂酶来治疗具有神经退行性疾病如帕金森病（PD）的受试者。可以例如通过基因治疗或通过施用葡糖脑苷脂酶蛋白来提供该酶。因此，本发明包括用于治疗PD或其它神经变性疾病的葡糖脑苷脂酶核酸或蛋白质。

4. 发明申请

WO2015069736A1 METHODS FOR EFFICIENT GENERATION OF GABAergic INTERNEURONS FROM PLURIPOTENT STEM CELLS 审中-公开
标题翻译：来自PLUIPOTENT干细胞的GABA能间质细胞的有效产生的方法
公开(公告)号：WO2015069736A1
公开(公告)日：2015-05-14
申请号：PCT/US2014/064085
申请日：2014-11-05
申请人： THE MCLEAN HOSPITAL CORPORATION
发明人： CHUNG, Sangmi
IPC分类号： A61P25/00
CPC分类号： C12N5/0619 , A61K35/30 , C12N2501/115 , C12N2501/119 , C12N2501/13 , C12N2501/155 , C12N2501/41 , C12N2501/415 , C12N2506/02 , C12N2506/08 , C12N2506/45
摘要： Enhanced methods for the generation of medial ganglionic eminence (MGE) cells from pluripotent stem cells are provided that involve an additional step of contacting the cells with an activator of FGF8 signaling while differentiating Pax6+ cells progenitor cells into MGE cells with an activator of sonic hedgehog, and optionally a Wnt inhibitor. The activator of FGF8 signaling shifts the differentiation of the population of cells to NKX2.1+ MGE cells, rather than to CopuTFII+ caudal ganglionic eminence (CGE) cells. Methods for reatment of neurological disorders, such as epilepsy, by transplant of MGE cells, or GABAergic interneurons derived from human pluripotent stem cells, into a subject in need of treatment are also provided. Human pluripotent stem cell derived MGE cells when transplanted successfully suppress spontaneous seizures, e.g. in epilepsy. We also have developed a method to purify MGE cells and maturing interneurons from differentiated pluripotent stem cells using cell surface marker and molecular beacon technology.
摘要翻译：提供了用于从多能干细胞产生内侧神经节隆突（MGE）细胞的增强方法，该方法涉及使细胞与FGF8信号传导物接触的另外步骤，同时用Ponic6刺激素激活剂将Pax6 +细胞祖细胞分化成MGE细胞，和任选的Wnt抑制剂。 FGF8信号的激活剂将细胞群的分化转移到NKX2.1 + MGE细胞，而不是CopuTFII +尾尾神经节隆起（CGE）细胞。还提供了将需要治疗的受试者的神经系统疾病如癫痫，通过移植MGE细胞或来自人多能干细胞的GABA能中间神经元进行复原的方法。人多能干细胞衍生的MGE细胞移植成功抑制自发性癫痫发作。在癫痫。我们还开发了一种使用细胞表面标记和分子信标技术从分化多能干细胞中纯化MGE细胞和成熟中间神经元的方法。

5. 发明申请

WO2012083128A2 PREVENTION AND TREATMENT OF DISEASES CHARACTERIZED BY MESENCEPHALIC DOPAMINERGIC NEURON CELL DEATH 审中-公开
标题翻译：麻疹多巴胺神经元细胞死亡特征预防和治疗疾病
公开(公告)号：WO2012083128A2
公开(公告)日：2012-06-21
申请号：PCT/US2011/065405
申请日：2011-12-16
申请人： THE MCLEAN HOSPITAL CORPORATION , ISACSON, Ole , OSBORN, Teresia
发明人： ISACSON, Ole , OSBORN, Teresia
CPC分类号： A61K31/381
摘要： Provided herein are methods for the prevention and treatment of diseases affecting mesencephalic dopaminergic neurons including, for example, Parkinson's disease. Suitable therapeutic agents for use in the methods described herein include, for example, agents that upregulate the expression En-1 and/or FoxA2 in target cells.
摘要翻译：本文提供了预防和治疗影响中脑多巴胺能神经元的疾病的方法，包括例如帕金森氏病。用于本文所述方法的合适的治疗剂包括例如在靶细胞中上调表达En-1和/或FoxA2的试剂。

6. 发明申请

WO2012074565A1 IMAGING-BASED IDENTIFICATION OF A NEUROLOGICAL DISEASE OR A NEUROLOGICAL DISORDER 审中-公开
标题翻译：基于成像的神经病或神经病学鉴别
公开(公告)号：WO2012074565A1
公开(公告)日：2012-06-07
申请号：PCT/US2011/022589
申请日：2011-01-26
申请人： UNIVERSITY OF UTAH RESEARCH FOUNDATION , THE MCLEAN HOSPITAL CORPORATION , LANGE, Nicholas, T. , LAINHART, Janet, E.
发明人： LANGE, Nicholas, T. , LAINHART, Janet, E.
IPC分类号： A61B5/055 , G06K9/36
CPC分类号： A61K45/00 , G06F19/00
摘要： System(s) and method(s) are provided to enable imaging-based identification, detection, evaluation, and mapping of white matter microstructure and hemispheric organization of white matter in a central nervous system (CNS) structure afflicted by a neurological disease or disorder. Various embodiments exploit a set of diffusion tensor metrics to define a classification sub-space and determine a multivariate classifier through training data related to at least two groups of subjects: a first group of subjects afflicted by the neurological disease or neurological disorder, and a second group of subjects typically developing. The set of diffusion tensor metrics can be selected based at least on clinical information related to the neurological disease or neurological disorder and anatomy of CNS structure. Inclusion of tensor skewness asymmetry in such set yields an increase in sensitivity, specificity, accuracy, reliability, and predictive ability of the biological discrimination of subjects with and without a neurological disease or neurological disorder.
摘要翻译：提供了系统和方法，以使基于成像的识别，检测，评估和绘制由神经系统疾病或病症所折磨的中枢神经系统（CNS）结构中的白质微结构和白质的半球组织。各种实施例利用一组扩散张量度量来定义分类子空间，并且通过与至少两组受试者相关的训练数据确定多变量分类器：受神经疾病或神经障碍所折磨的第一组受试者，以及第二组一组通常发展的科目。可以至少基于与神经系统疾病或神经系统疾病和CNS结构的解剖学有关的临床信息来选择一组扩散张量度量。在这种集合中包含张量偏度不对称性导致具有和不具有神经疾病或神经障碍的受试者的生物辨别的灵敏度，特异性，准确性，可靠性和预测能力的增加。

7. 发明申请

WO2011130675A2 DOPAMINERGIC NEURONS DIFFERENTIATED FROM PLURIPOTENT STEM CELLS AND USES OF THEREOF 审中-公开
标题翻译：由多种干细胞分化的多巴胺神经元及其用途
公开(公告)号：WO2011130675A2
公开(公告)日：2011-10-20
申请号：PCT/US2011/032745
申请日：2011-04-15
申请人： THE MCLEAN HOSPITAL CORPORATION , COOPER, Oliver , ISACSON, Ole
发明人： COOPER, Oliver , ISACSON, Ole
IPC分类号： C12N5/0735 , C12N5/079 , C12N5/02 , A61K35/30 , A61P25/16
CPC分类号： A61K35/30 , C12N5/0619 , C12N2500/38 , C12N2501/01 , C12N2501/119 , C12N2501/13 , C12N2501/15 , C12N2501/155 , C12N2501/41 , C12N2501/415 , C12N2506/02 , C12N2506/45
摘要： The present invention provides populations of neural cells derived from pluripotent cells, and methods for making and using the same. Disclosed herein are methods for generating dopaminergic neurons in vitro using a combination of agents that cause differentiation of the pluripotent cells into dopaminergic neurons. Also disclosed are methods for treating a neurodegenerative disease in a patient by generating dopaminergic neurons in vitro , and transplanting them into the brain of the patient, such that the dopaminergic neurons are sufficient to reduce or eliminate the symptoms of the neurodegenerative disease.
摘要翻译：本发明提供了源自多能细胞的神经细胞群体及其制备和使用方法。本文公开的是使用导致多能细胞分化成多巴胺能神经元的试剂组合在体外产生多巴胺能神经元的方法。还公开了通过在体外产生多巴胺能神经元并将其移植到患者的脑中来治疗患者的神经变性疾病的方法，使得多巴胺能神经元足以减少或消除神经变性疾病的症状。

8. 发明申请

WO2011090947A2 METHODS FOR THE DIAGNOSIS AND TREATMENT OF PARKINSON'S DISEASE 审中-公开
标题翻译：方法诊断和治疗帕金森病
公开(公告)号：WO2011090947A2
公开(公告)日：2011-07-28
申请号：PCT/US2011/021581
申请日：2011-01-18
申请人： THE MCLEAN HOSPITAL CORPORATION , CHUNG, Chee Yeun , ISACSON, Ole
发明人： CHUNG, Chee Yeun , ISACSON, Ole
IPC分类号： A61K31/7105 , A61K38/00
CPC分类号： A61K38/1709
摘要： The present invention relates to methods for treatment or prevention of Parkinsons disease and associated conditions by increasing the expression Otx2 in the midbrain dopaminergic neurons of the patient. The present invention further relates to methods for the diagnosis and evaluation of Parkinsons disease. For instance, patient test samples are analyzed for the presence of one or more specific variant sequences associated with Parkinsons disease. In some embodiments, the variant is a promoter mutation in the Otx2 gene.
摘要翻译：本发明涉及通过增加患者的中脑多巴胺能神经元中的Otx2表达来治疗或预防帕金森病和相关病症的方法。本发明还涉及用于诊断和评估帕金森病的方法。例如，分析患者测试样品中与Parkinsons疾病相关的一个或多个特异性变体序列的存在。在一些实施方案中，该变体是Otx2基因中的启动子突变。

9. 发明申请

WO2009152407A1 THERAPEUTIC IMPRINTING FOR THE TREATMENT OF PSYCHIATRIC DISORDERS 审中-公开
标题翻译：治疗心理障碍的治疗方法
公开(公告)号：WO2009152407A1
公开(公告)日：2009-12-17
申请号：PCT/US2009/047158
申请日：2009-06-12
申请人： THE MCLEAN HOSPITAL CORPORATION , ANDERSEN, Susan, L.
发明人： ANDERSEN, Susan, L.
IPC分类号： A01N45/00 , A61K31/59
CPC分类号： A61K31/59
摘要： The invention features methods, kits, and compositions for the treatment of psychiatric disorders using therapeutic imprinting in pre-pubescent subjects.
摘要翻译：本发明的特征在于使用治疗性印记在前短柔毛受试者中治疗精神疾病的方法，试剂盒和组合物。

10. 发明申请

WO2007127393A2 TREATMENT AND SCREENING METHODS FOR PROMOTING NEUROGENESIS 审中-公开
标题翻译：治疗和筛选方法促进神经发生
公开(公告)号：WO2007127393A2
公开(公告)日：2007-11-08
申请号：PCT/US2007/010284
申请日：2007-04-27
申请人： THE MCLEAN HOSPITAL CORPORATION , CARLEZON, William, A., Jr.
发明人： CARLEZON, William, A., Jr.
IPC分类号： A61K48/00 , A61K39/395 , C12Q1/30
CPC分类号： G01N33/566 , C07K14/4703 , C12N15/113 , C12N2310/14 , G01N33/6896 , G01N2800/28 , G01N2800/30
摘要： The invention features methods for promoting neurogenesis including treatment of psychiatric disorders (e.g., depression, bipolar disorder, and post traumatic stress disorder), drug abuse or addiction, neurodegenerative diseases (e.g., Alzheimer's disease, Parkinson's disease, amyotrophic lateral sclerosis, multiple sclerosis, frontotemporal dementia, Huntington's disease, and prion disease), and head trauma (e.g., stroke and physical injury) by inhibition of Sprouty (SPRY) and methods for identification of compounds useful promoting neurogenesis (e.g., useful in the treatment of psychiatric disorders, drug abuse or addiction, neurodegenerative diseases, or head trauma).
摘要翻译：本发明涉及用于促进神经发生的方法，包括治疗精神疾病（例如抑郁症，双相性精神障碍和创伤后应激障碍），药物滥用或成瘾，神经变性疾病（例如阿尔茨海默病，帕金森病，肌萎缩性侧索硬化，多发性硬化，额颞叶痴呆，亨廷顿舞蹈病和朊病毒疾病），以及通过抑制Sprouty（SPRY）的头部创伤（例如中风和身体伤害）和用于鉴定促进神经发生的化合物的方法（例如，用于治疗精神疾病，药物滥用或成瘾，神经变性疾病或头部创伤）。

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