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1. 发明授权

US09696297B2 Method for preparing an X chromosome inactivated female human neural cell 有权
公开(公告)号：US09696297B2
公开(公告)日：2017-07-04
申请号：US13518842
申请日：2010-12-23
申请人： Maria C. N. Marchetto , Fred H. Gage , Alysson R. Muotri
发明人： Maria C. N. Marchetto , Fred H. Gage , Alysson R. Muotri
IPC分类号： C12N5/079 , G01N33/50
CPC分类号： G01N33/5058 , G01N2800/28
摘要： Provided herein are methods and compositions useful in treating neurological disorders.

2. 发明申请

US20130315886A1 RETROELEMENTS AND MENTAL DISORDERS AND METHODS OF MEASURING L1 RETROTRANSPOSITION 审中-公开
标题翻译：退化和精神障碍及其测量方法
公开(公告)号：US20130315886A1
公开(公告)日：2013-11-28
申请号：US13883962
申请日：2010-08-04
申请人： Fred H. Gage , Nicole Coufal , Michael J. McConnell , Alysson Muotri , Maria C.N. Marchetto
发明人： Fred H. Gage , Nicole Coufal , Michael J. McConnell , Alysson Muotri , Maria C.N. Marchetto
IPC分类号： C12Q1/68
CPC分类号： C12Q1/6883 , A61K31/713 , A61K48/00 , C12Q2600/106 , C12Q2600/154 , C12Q2600/156 , C12Q2600/158 , C12Q2600/16 , G01N33/5058 , G01N33/6896 , G01N2800/28
摘要： A method of treating increased non-LTR retrotransposition in a cell. The method includes exposing a neural cell to a retrotransposition inhibitor in an amount sufficient to decrease the non-LTR retrotransposition in the neural cell or a progeny of the neural cell. In various embodiments, the non-LTR retrotransposition involves at least one L1 retrotransposon. Also provided is a method of assaying retrotransposition in neural cells. The method includes sorting synchronized neural cells of the same genetic background into single neural cells, and subjecting one or more of the sorted single neural cells to quantitative polymerase chain reaction amplification of at least one retrotransposon. In addition, a method of identifying an inhibitor of retrotransposition and a identifying a neural condition associated with non-LTR retrotransposition are provided.
摘要翻译：治疗细胞中增加的非LTR逆转录的方法。该方法包括以足以减少神经细胞或神经细胞的后代中的非LTR逆转录的量将神经细胞暴露于逆转录抑制剂。在各种实施方案中，非LTR反转录涉及至少一个L1反转录转座子。还提供了测定神经细胞中的逆转录的方法。该方法包括将相同遗传背景的同步神经细胞分选成单个神经细胞，并使一个或多个分选的单个神经细胞经受至少一个反转录转座子的定量聚合酶链反应扩增。此外，提供了鉴定逆转录抑制剂的方法和鉴定与非LTR逆转录相关的神经病症。

3. 发明申请

US20120129835A1 SCHIZOPHRENIA METHODS AND COMPOSITIONS 审中-公开
标题翻译： SCHIZOPHRENIA方法和组合物
公开(公告)号：US20120129835A1
公开(公告)日：2012-05-24
申请号：US13298246
申请日：2011-11-16
申请人： Kristen Brennand , Fred H. Gage
发明人： Kristen Brennand , Fred H. Gage
IPC分类号： A61K31/553 , C12Q1/06 , C40B30/04 , C12Q1/68 , A61K31/5415 , C12Q1/70 , A61P25/18 , A61K31/5513 , A61K31/519 , G01N33/566 , C12Q1/02
CPC分类号： G01N33/5058 , A61K31/519 , A61K31/5415 , A61K31/5513 , A61K31/553 , C12Q1/6883 , C12Q2600/158 , G01N33/5091 , G01N2800/302 , G01N2800/52
摘要： Methods of preparing and using neural cells derived from human induced pluripotent stem cell (hiPSCs), particularly hiPSCs derived from subjects with schizophrenia are provided. The hiPSC-derived neural cells can be used to screen test compounds and to identify schizophrenia marker functions. The hiPSC-derived neural cells can be used to diagnose and/or assess the severity of schizophrenia in a subject. Further, may the hiPSC-derived neural cells from a subject be used as an in vitro system to identify the most effective candidate among existing drugs for that specific subject (i.e. personalized medicine).
摘要翻译：提供了制备和使用衍生自人诱导多能干细胞（hiPSC）的神经细胞，特别是源自精神分裂症受试者的hiPSC的方法。 hiPSC衍生的神经细胞可用于筛选测试化合物并鉴定精神分裂症标记功能。 hiPSC衍生的神经细胞可用于诊断和/或评估受试者中精神分裂症的严重程度。此外，可以将来自受试者的来自hiPSC的神经细胞用作体外系统，以鉴定该特定受试者（即，个体化药物）的现有药物中最有效的候选物。

4. 发明申请

US20080227829A1 Neurogenic compounds 审中-公开
标题翻译：神经源性化合物
公开(公告)号：US20080227829A1
公开(公告)日：2008-09-18
申请号：US12069861
申请日：2008-02-13
申请人： John F. Hammerstone , Mark A. Kelm , Fred H. Gage , Henriette van Praag
发明人： John F. Hammerstone , Mark A. Kelm , Fred H. Gage , Henriette van Praag
IPC分类号： A61K31/44 , A61K31/35 , A61P25/00
CPC分类号： A61K31/35 , A61K31/44 , A61K31/70
摘要： The invention relates to method(s) of use of the compound(s) described herein, e.g. method for stimulating neurogenesis, including in vitro neurogenesis, by contacting neuronal progenitor cells with an effective amount of the compound(s) described herein; method for treatment of a subject in need of treatment with a neurogenic compound; and/or for treatment of a disease or condition associated with damage to the hippocampus. The subject may be a human or a veterinary animal.
摘要翻译：本发明涉及本文所述的化合物的使用方法，例如，通过使神经祖细胞与有效量的本文所述的化合物接触来刺激神经发生的方法，包括体外神经发生，用于治疗需要用神经源性化合物治疗的受试者的方法; 和/或用于治疗与海马损伤相关的疾病或病症。受试者可以是人类或兽医动物。

5. 发明授权

US06326484B1 Nucleic acids encoding regulators of FGF-2 transcription (RFT) and variants thereof 失效
标题翻译：编码FGF-2转录调节因子（RFT）的核酸及其变体
公开(公告)号：US06326484B1
公开(公告)日：2001-12-04
申请号：US09079431
申请日：1998-05-14
申请人： Fred H. Gage , Tetsuya Ueba
发明人： Fred H. Gage , Tetsuya Ueba
IPC分类号： C07H2104
CPC分类号： C07K14/4702 , A01K2217/05 , A61K38/00
摘要： The present invention is based on the discovery of genes which encode polypeptides are FGF-2 regulators of transcription (RFT). RFT-A, a negative regulator of transcription and RFT-A′ and RFT-B, positive regulators of FGF-2 traniscriptionl are provided. Also included are methods for diagnosis of prognosis for a subject having or at risk of having a disorder associated with FGF-2 and for treatment of cell proliferative disorders associated with FGF-2. Screening methods for identifying a compound which affects RTT-A polypeptide or a variant of RFT-A polypeptide and for identifying proteins that bind to RFT-A polypeptide or a variant of RFT-A are also provided.
摘要翻译：本发明基于编码多肽的基因的发现是FGF-2转录调节因子（RFT）。提供RFT-A，转录的负调节子和RFT-A'和RFT-B，FGF-2转录物的阳性调节物。还包括用于诊断具有或具有与FGF-2相关的疾病或具有与FGF-2相关的细胞增殖性疾病的风险的受试者的预后的方法。还提供了鉴定影响RTT-A多肽或RFT-A多肽的变体并鉴定结合RFT-A多肽或RFT-A的变体的蛋白质的化合物的筛选方法。

6. 发明授权

US5674486A Cancer immunotherapy with carrier cells 失效
标题翻译：用载体细胞进行癌症免疫治疗
公开(公告)号：US5674486A
公开(公告)日：1997-10-07
申请号：US473123
申请日：1995-06-06
申请人： Robert E. Sobol , Fred H. Gage , Ivor Royston , Theodore Friedmann
发明人： Robert E. Sobol , Fred H. Gage , Ivor Royston , Theodore Friedmann
IPC分类号： A61K38/19 , A61K38/20 , A61K39/00 , A61K48/00 , A61K35/76 , C12N5/00
CPC分类号： A61K38/2013 , A61K39/0011 , A61K48/00
摘要： A novel method of tumor immunotherapy is described comprising the genetic modification of cells resulting in the secretion of cytokine gene products to stimulate a patient's immune response to tumor antigens. In one embodiment, autologous fibroblasts genetically modified to secrete at least one cytokine gene product are utilized to immunize the patient in a formulation with tumor antigens at a site other than an active tumor site. In another embodiment, cells genetically modified to express at least one tumor antigen gene product and to secrete at least one cytokine gene product are utilized in a formulation to immunize the patient at a site other than an active tumor site.
摘要翻译：描述了一种新的肿瘤免疫治疗方法，其包括导致分泌细胞因子基因产物以刺激患者对肿瘤抗原的免疫应答的细胞的遗传修饰。在一个实施方案中，利用经遗传修饰以分泌至少一种细胞因子基因产物的自体成纤维细胞在具有肿瘤抗原的制剂中在除活性肿瘤部位之外的部位免疫患者。在另一个实施方案中，在制剂中利用遗传修饰以表达至少一种肿瘤抗原基因产物并分泌至少一种细胞因子基因产物的细胞，以在除活性肿瘤部位之外的部位免疫患者。

7. 发明授权

US5082670A Method of grafting genetically modified cells to treat defects, disease or damage or the central nervous system 失效
标题翻译：研究遗传改良细胞治疗缺陷，疾病或损害或中枢神经系统的方法
公开(公告)号：US5082670A
公开(公告)日：1992-01-21
申请号：US285196
申请日：1988-12-15
申请人： Fred H. Gage , Michael B. Rosenberg , Theodore Friedmann
发明人： Fred H. Gage , Michael B. Rosenberg , Theodore Friedmann
IPC分类号： A61K35/12 , A61K38/00 , A61K38/18 , A61K38/44 , A61K48/00 , C07K14/48 , C12N9/02 , C12N9/10 , C12N15/867
CPC分类号： C12N15/86 , A61K38/1825 , A61K48/00 , C07K14/48 , C12N9/0071 , C12N9/1029 , C12Y114/16002 , A61K38/00 , C12N2740/13043 , Y10S435/948
摘要： Methods of genetically modifying donor cells by gene transfer for grafting into the central nervous system to treat diseased or damaged cells are disclosed. The modified donor cells produce a molecule capable of affecting the recovery of cells in the CNS. Methods and vectors for carrying out gene transfer and grafting are described.
摘要翻译：公开了通过基因转移遗传修饰供体细胞用于接枝入中枢神经系统以治疗患病或受损细胞的方法。修饰的供体细胞产生能够影响CNS细胞恢复的分子。描述了用于进行基因转移和接枝的方法和载体。

8. 发明授权

US08092992B2 Transcriptional regulation of gene expression by small double-stranded modulatory RNA 有权
标题翻译：通过小双链调节RNA的基因表达的转录调控
公开(公告)号：US08092992B2
公开(公告)日：2012-01-10
申请号：US10857784
申请日：2004-05-28
申请人： Tomoko Kuwabara , Fred H. Gage
发明人： Tomoko Kuwabara , Fred H. Gage
IPC分类号： C12Q1/68 , C12N5/00 , C12N5/02 , A61K48/00 , C07H21/04 , C07H21/02
CPC分类号： C12N5/0619 , C12N2501/60 , C12N2799/027
摘要： The invention provides a method for modulating gene expression by contacting a cellular system with a double-stranded ribonucleic acid molecule capable of associating with a regulatory machinery that controls transcription of one or more genes, wherein the association results in altered expression of the one or more genes. The invention is further directed to method for directing the differentiation of neuronal stem cells into neurons by contacting a cellular system with a double-stranded ribonucleic acid molecule capable of associating with a regulatory machinery that controls transcription of one or more genes involved in neuronal differentiation and directing the transcription of the one or more genes. In related embodiments, the invention provides particular compositions of double-stranded ribonucleic acid molecules as well as therapeutic and screening applications of the invention.
摘要翻译：本发明提供了一种通过使细胞系统与能够与控制一种或多种基因转录的调节机制缔合的双链核糖核酸分子来调节基因表达的方法，其中所述缔合导致一个或多个基因。本发明还涉及通过使细胞系统与能够与控制一种或多种参与神经元分化的基因转录的调节机制相关联的双链核糖核酸分子的方式来指导神经元干细胞分化为神经元的方法并指导一个或多个基因的转录。在相关实施方案中，本发明提供了双链核糖核酸分子的特定组合物以及本发明的治疗和筛选应用。

9. 发明申请

US20100235310A1 Temporally dynamic artificial neural networks 有权
标题翻译：时间动态人工神经网络
公开(公告)号：US20100235310A1
公开(公告)日：2010-09-16
申请号：US12657748
申请日：2010-01-27
申请人： Fred H. Gage , James Bradley Aimone , Janet Wiles
发明人： Fred H. Gage , James Bradley Aimone , Janet Wiles
IPC分类号： G06F15/18 , G06N3/02
CPC分类号： G06N3/082
摘要： An apparatus, article and method containing an artificial neural network that, after training, produces new trainable nodes such that input data representative of a first event and input data representative of a second event both activate a subset of the new trainable nodes. The artificial neural network can generate an output that is influenced by the input data of both events. In various embodiments, the new trainable nodes are sequentially produced and show decreasing trainability over time such that, at a particular point in time, newer produced nodes are more trainable than earlier produced nodes. The artificial neural network can be included in various embodiments of methods, apparatus and articles for use in predicting or profiling events.
摘要翻译：一种包含人造神经网络的装置，物品和方法，其在训练之后产生新的可训练节点，使得表示第一事件的输入数据和表示第二事件的输入数据都激活新的可训练节点的子集。人造神经网络可以产生受两个事件的输入数据影响的输出。在各种实施例中，新的可训练节点被顺序地产生并且随着时间的推移显示降低的可训练性，使得在特定的时间点，较新的生成的节点比先前产生的节点更可训练。人造神经网络可以包括在用于预测或分析事件的方法，装置和物品的各种实施例中。

10. 发明申请

US20100184152A1 TARGET-ORIENTED WHOLE GENOME AMPLIFICATION OF NUCLEIC ACIDS 审中-公开
标题翻译：目标靶向的核酸的全基因组扩增
公开(公告)号：US20100184152A1
公开(公告)日：2010-07-22
申请号：US12445926
申请日：2007-10-22
申请人： Vladislav Sandler , Fred H. Gage
发明人： Vladislav Sandler , Fred H. Gage
IPC分类号： C12P19/34 , C12N9/10
CPC分类号： C12Q1/6846 , C12Q2531/119 , C12Q2525/179 , C12Q2525/161
摘要： Disclosed herein are methods of amplifying target nucleic acid sequences (e.g., DNA or RNA), particularly from a very small amount of starting material, such as a single cell. These methods involve targeting the amplification of specific sequence(s) by use of sequence-specific primers and random primers for whole genome amplification using multiple displacement amplification. Generally, the provided methods are referred to herein as “target-oriented” whole genome amplification. Starting material for target-oriented whole genome amplification can be any sample containing DNA or RNA, however, the technique is particularly suitable for very small amounts of starting material, such as a few cells, a single cell, or a single nucleus. The methods provide amplified nucleic acid (including the target sequence of interest) that can subsequently be analyzed.
摘要翻译：本文公开了扩增靶核酸序列（例如DNA或RNA）的方法，特别是从非常少量的起始材料如单细胞扩增的方法。这些方法包括通过使用序列特异性引物和使用多重置换扩增的全基因组扩增的随机引物靶向特异性序列的扩增。通常，所提供的方法在本文中称为“靶向”全基因组扩增。用于靶向全基因组扩增的起始材料可以是含有DNA或RNA的任何样品，然而，该技术特别适用于非常少量的起始材料，例如少量细胞，单细胞或单核。该方法提供随后可以分析的扩增核酸（包括目标靶序列）。

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