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1. 发明申请

WO2019118325A1 ANTISENSE COMPOUNDS TARGETING LEUCINE-RICH REPEAT KINASE 2 (LRRK2) FOR THE TREATMENT OF PARKINSONS DISEASE 审中-公开
公开(公告)号：WO2019118325A1
公开(公告)日：2019-06-20
申请号：PCT/US2018/064693
申请日：2018-12-10
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
CPC分类号： C12N9/12 , C12N15/1137 , C12N2310/11 , C12N2310/3233 , C12N2320/33 , C12Y207/11001
摘要： The present disclosure relates generality to compounds comprising oligonucleotides complementary to Leucine-Rich-Repeat-Kinase (LRRK2) RNA transcript Certain such compounds are useful for hybridising 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.

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

WO2012048330A3 TREATMENT OF MOTOR NEURON DISEASE 审中-公开
公开(公告)号：WO2012048330A3
公开(公告)日：2012-04-12
申请号：PCT/US2011/055645
申请日：2011-10-10
申请人： THE MCLEAN HOSPITAL CORPORATION , ISACSON, Ole , OSBORN, Anna Charlotta Teresia Magnuson
发明人： ISACSON, Ole , OSBORN, Anna Charlotta Teresia Magnuson
IPC分类号： A61K31/404 , A61K31/403 , A61K31/165 , A61K31/16 , A61P25/28 , A61P25/00
摘要： Provided herein are methods and compositions for the treatment of motor neuron diseases including, for example, amyotrophic lateral sclerosis. Suitable therapeutic agents include, for example, agents that up-regulate the expression IGF-II or guanine deaminase in a cell.

4. 发明申请

WO2016138392A1 METHODS AND COMPOSITIONS FOR THE TREATMENT OR PREVENTION OF PARKINSON'S DISEASE 审中-公开
标题翻译：用于治疗或预防帕金森病的方法和组合物
公开(公告)号：WO2016138392A1
公开(公告)日：2016-09-01
申请号：PCT/US2016/019798
申请日：2016-02-26
申请人： THE MCLEAN HOSPITAL CORPORATION
发明人： ISACSON, Ole
IPC分类号： A01N63/00 , C12N5/02 , C12N5/071
CPC分类号： A61K35/30 , A61K38/185 , C12N5/0619 , C12N2506/45
摘要： Provided are methods and compositions for the treatment and/or prevention of Parkinson's disease in humans. Specifically, the compositions comprising: (a) a population of cells comprising at least 3% dopaminergic neurons, wherein the dopaminergic neurons express FOXA2, Beta-tubulin, and tyrosine hydroxylase (TH), (b) a neurotrophic factor, and (c) a phannaceutically acceptable carrier; and methods for the treatment and/or prevention of Parkinson's disease comprising administering or transplanting a composition comprising dopaminergic neurons derived from the patient's own cells.
摘要翻译：提供了用于治疗和/或预防人类帕金森病的方法和组合物。具体而言，所述组合物包含：（a）包含至少3％多巴胺能神经元的细胞群，其中所述多巴胺能神经元表达FOXA2，β-微管蛋白和酪氨酸羟化酶（TH），（b）神经营养因子，和（c）药物可接受的载体; 以及用于治疗和/或预防帕金森病的方法，包括施用或移植包含源于患者自身细胞的多巴胺能神经元的组合物。

5. 发明申请

WO2003033463A3 MULTIMERIZED ENHANCER DOMAINS FOR CELL-SPECIFIC EXPRESSION 审中-公开
公开(公告)号：WO2003033463A3
公开(公告)日：2003-04-24
申请号：PCT/US2002/033790
申请日：2002-10-21
申请人： THE MCLEAN HOSPITAL CORPORATION , KIM, Kwang-Soo , ISACSON, Ole , HWANG, Dong-Youn
发明人： KIM, Kwang-Soo , ISACSON, Ole , HWANG, Dong-Youn
IPC分类号： C12N15/63
摘要： The invention features an enhancer cassette and methods of its use. The enhancer cassette has the formula [X-Y] n , wherein each X is independently a cell type-specific enhancer element; Y is absent or is a mono or polynucleotide that has between one and thirty nucleotides; and n is an integer between five and fifty, inclusive.

6. 发明申请

WO2004053084A2 DOPAMINERGIC NEURONS DIFFERENTIATED FROM EMBRYONIC CELLS FOR TREATING NEURODEGENERATIVE DISEASES 审中-公开
标题翻译：用于治疗神经损伤性疾病的胚胎细胞分化的多巴胺神经元
公开(公告)号：WO2004053084A2
公开(公告)日：2004-06-24
申请号：PCT/US2003/038919
申请日：2003-12-09
申请人： THE MCLEAN HOSPITAL CORPORATION
发明人： ISACSON, Ole , BJÖRKLUND, Lars
IPC分类号： C12N
CPC分类号： C12N5/0619 , C12N2501/15 , C12N2501/40 , C12N2506/02
摘要： Disclosed herein are methods for generating dopaminergic neurons in vitro by inhibiting a pathway component of a TGF-β signaling pathway and overexpressing one or more cell fate-inducing polypeptides in pluripotent cells, causing 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 neurodegerative disease.
摘要翻译：本文公开了通过抑制TGF-β信号传导途径的途径组分并在多能细胞中过表达一种或多种细胞命运诱导多肽，导致多能细胞分化成多巴胺能神经元的方法来体外产生多巴胺能神经元。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。

7. 发明申请

WO1996014399A1 PORCINE STRIATAL CELLS AND THEIR USE IN TREATMENT OF NEUROLOGICAL DEFICITS DUE TO NEURODEGENERATIVE DISEASE 审中-公开
标题翻译： PORCINE STRIATAL细胞及其用于治疗神经病变疾病的神经缺陷的用途
公开(公告)号：WO1996014399A1
公开(公告)日：1996-05-17
申请号：PCT/US1995014453
申请日：1995-11-07
申请人： DIACRIN, INC. , THE MCLEAN HOSPITAL CORPORATION
发明人： DIACRIN, INC. , THE MCLEAN HOSPITAL CORPORATION , ISACSON, Ole , DINSMORE, Jonathan
IPC分类号： C12N05/06
CPC分类号： C12N5/0618 , A61K35/30 , A61K38/13 , A61K39/001 , A61K2035/122 , C07K16/2833 , C07K2317/54 , Y10S424/809 , A61K2300/00
摘要： Porcine striatal cells and methods for using the cells to treat neurological deficits due to neurodegeneration are described. The porcine striatal cells can be modified to be suitable for transplantation into a xenogeneic subject, such as a human. For example, the porcine striatal cells can be modified such that an antigen (e.g., an MHC class I antigen) on the cell surface which is capable of stimulating an immune response against the cell in a xenogeneic subject is altered (e.g., by contact with an anti-MHC class I antibody, or a fragment or derivative thereof) to inhibit rejection of the cell when introduced into the subject. In one embodiment, the porcine striatal cells are obtained from a pig which is essentially free from organisms or substances which are capable of transmitting infection or disease to the recipient subject. The porcine striatal cells of the present invention can be used to treat neurological deficits due to neurodegeneration in the brain of a xenogeneic subject (e.g., a human with epilepsy, head trauma, stroke, amyotrophic lateral sclerosis, Parkinson's disease, Alzheimer's disease, or Huntington's disease) by introducing the cells into the brain of the subject.
摘要翻译：描述了猪纹状体细胞和使用细胞治疗由于神经变性引起的神经缺陷的方法。猪纹状体细胞可以被修饰为适合于移植到异种受试者（例如人）中。例如，可以修饰猪纹状体细胞，使得在异种受试者中能够刺激针对细胞的免疫应答的细胞表面上的抗原（例如MHC I类抗原）被改变（例如，通过与抗MHC I类抗体或其片段或衍生物），以抑制当引入受试者时细胞的排斥反应。在一个实施方案中，猪纹状体细胞从基本上不含能够将感染或疾病传播给接受者的生物或物质的猪获得。本发明的猪纹状体细胞可用于治疗异种受试者的脑部神经变性（例如，患有癫痫，头部创伤，中风，肌萎缩性侧索硬化，帕金森病，阿尔茨海默病或亨廷顿病疾病）通过将细胞引入受试者的脑中。

8. 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。

9. 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。

10. 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。

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