公开(公告)号：US20160270377A1

公开(公告)日：2016-09-22

申请号：US15072286

申请日：2016-03-16

申请人： Regeneron Pharmaceuticals, Inc.

发明人： Burcin Ikiz ,  Michael LaCroix-Fralish ,  Susan D. Croll

IPC分类号： A01K67/027 ,  G01N33/50

CPC分类号： A01K67/0276 ,  A01K67/0278 ,  A01K2217/075 ,  A01K2217/077 ,  A01K2227/105 ,  A01K2267/0318 ,  A01K2267/0393 ,  C07K14/70578 ,  C07K2319/02 ,  C07K2319/03 ,  C07K2319/61 ,  G01N33/5058 ,  G01N33/5088 ,  G01N2500/00 ,  G01N2800/28

摘要： An animal model for motor neuron dysfunction in disease, e.g., amyotrophic lateral sclerosis (ALS), comprising a genetically modified non-human animal that comprises a genetically modified DR6 allele and exhibits normal phenotypes at birth and for a few weeks or months after birth. However, as the non-human animal ages, it develops motor neuron dysfunction that presents as one or more ALS-like symptoms, which may progress rapidly after onset. Methods of identifying candidate agents that may be used to prevent, delay or treat ALS are also provided.

摘要翻译： 疾病运动神经元功能障碍的动物模型，例如肌萎缩性侧索硬化（ALS），其包含遗传修饰的非人动物，其包含遗传修饰的DR6等位基因，并且在出生时和出生后数周或数月内呈现正常表型。 然而，随着非人类动物年龄的增长，它产生运动神经元功能障碍，其呈现为一种或多种类似ALS的症状，其在发病后可能快速进展。 还提供了可用于预防，延迟或治疗ALS的候选药物的鉴定方法。

公开(公告)号：US09421258B2

公开(公告)日：2016-08-23

申请号：US14555048

申请日：2014-11-26

申请人： The Board of Trustees of the Leland Stanford Junior University

发明人： Karl Deisseroth ,  Kay Tye ,  Lief Fenno

IPC分类号： A61N5/06 ,  A61K41/00 ,  A01K67/027 ,  A61K49/00 ,  A61B5/16 ,  A61K38/17

CPC分类号： A61K38/177 ,  A01K67/0275 ,  A01K67/0278 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/0356 ,  A01K2267/0393 ,  A61B5/165 ,  A61K9/0019 ,  A61K41/00 ,  A61K48/0058 ,  A61K49/0004 ,  A61K49/0008 ,  A61N5/062 ,  C07K14/705 ,  C12N7/00 ,  C12N15/8509 ,  C12N15/86 ,  C12N2015/859 ,  C12N2750/14143 ,  C12N2799/025 ,  G01N33/5088

摘要： Provided herein are animals expressing light-responsive opsin proteins in the basal lateral amygdala of the brain and methods for producing the same wherein illumination of the light-responsive opsin proteins causes anxiety in the animal. Also provided herein are methods for alleviating and inducing anxiety in an animal as well as methods for screening for a compound that alleviates anxiety in an animal.

公开(公告)号：US20160237126A1

公开(公告)日：2016-08-18

申请号：US15147772

申请日：2016-05-05

申请人： The Board of Trustees of the Leland Stanford Junior University

发明人： Karl Deisseroth ,  Ofer Yizhar ,  Lief Fenno ,  Peter Hegemann ,  Matthias Prigge

IPC分类号： C07K14/405 ,  C12N13/00 ,  G01N33/50 ,  A01K67/027

CPC分类号： C07K14/405 ,  A01K67/0275 ,  A01K2217/052 ,  A01K2227/105 ,  A01K2267/0393 ,  A61K38/00 ,  A61N5/06 ,  A61N2005/0663 ,  C07K19/00 ,  C07K2319/00 ,  C07K2319/60 ,  C12N13/00 ,  G01N33/5058 ,  G01N2500/10

摘要： Provided herein are compositions comprising light-activated chimeric proteins expressed on plasma membranes and methods of using the same to selectively depolarize excitatory or inhibitory neurons.

摘要翻译： 本文提供了包含在质膜上表达的光激活嵌合蛋白质的组合物以及使用其来选择性去极化兴奋性或抑制性神经元的方法。

公开(公告)号：US20160219844A1

公开(公告)日：2016-08-04

申请号：US15021026

申请日：2014-09-12

申请人： ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI

发明人： Brian BROWN ,  Judith AGUDO

IPC分类号： A01K67/027 ,  G01N33/566 ,  C07K14/725

CPC分类号： A01K67/0275 ,  A01K2217/30 ,  A01K2227/105 ,  A01K2267/0387 ,  A01K2267/0393 ,  C07K14/7051 ,  C12N15/8509 ,  G01N33/566 ,  G01N2333/70503

摘要： This invention relates to a transgenic non-human mammal whose genome comprises a polynucleotide sequence encoding a T cell receptor that is specific to a fluorescent protein, where the T cell of the non-human mammal comprises the T cell receptor. The present invention also relates to an isolated T cell from the transgenic non-human mammal of the present invention, an isolated T cell comprising an expression construct comprising a polynucleotide sequence that encodes a T cell receptor that is specific to a fluorescent protein, methods of making transgenic non-human mammals comprising T cell receptors that are specific to a fluorescent protein, a method of depleting cells in a non-human mammal using isolated T cells that encode a T cell receptor that is specific to a target protein, and a method of characterizing a T cell response to an agent.

摘要翻译： 本发明涉及一种转基因非人哺乳动物，其基因组包含编码对荧光蛋白特异性的T细胞受体的多核苷酸序列，其中非人哺乳动物的T细胞包含T细胞受体。 本发明还涉及本发明的转基因非人哺乳动物的分离的T细胞，包含含有编码对荧光蛋白特异的T细胞受体的多核苷酸序列的表达构建体的分离的T细胞， 制备包含对荧光蛋白特异性的T细胞受体的转基因非人哺乳动物，使用编码靶蛋白特异性的T细胞受体的分离的T细胞消耗非人哺乳动物细胞的方法，以及方法 表征T细胞对药剂的反应。

公开(公告)号：US20160199663A1

公开(公告)日：2016-07-14

申请号：US14911405

申请日：2014-08-13

申请人： THE BOARD OF TRUSTEES OF THE LELAND STANFORD JUNIOR UNIVERSITY ,  CIRCUIT THERAPEUTICS, INC.

发明人： Shrivats Mohan Iyer ,  Scott L. Delp ,  Kathryn L. Montgomery ,  Karl A. Deisseroth ,  Christopher Towne

IPC分类号： A61N5/06 ,  C12N15/85 ,  A61K48/00 ,  A01K67/027 ,  A61K38/16 ,  A61K41/00

CPC分类号： A61N5/062 ,  A01K67/0275 ,  A01K67/0278 ,  A01K2207/15 ,  A01K2217/07 ,  A01K2217/072 ,  A01K2227/105 ,  A01K2267/03 ,  A01K2267/0393 ,  A61K38/16 ,  A61K41/00 ,  A61K48/0058 ,  A61K48/0075 ,  A61N5/0622 ,  C12N15/8509 ,  C12N2015/8527 ,  C12N2750/14141

摘要： The present disclosure provides compositions and methods for controlling pain. The present disclosure provides methods for identifying agents that control pain.

摘要翻译： 本公开提供了用于控制疼痛的组合物和方法。 本公开提供了用于鉴定控制疼痛的药剂的方法。

公开(公告)号：US20160176919A1

公开(公告)日：2016-06-23

申请号：US14978700

申请日：2015-12-22

申请人： Michael Cohen ,  Ruth Barrett ,  Haihong Jin

发明人： Michael Cohen ,  Ruth Barrett ,  Haihong Jin

IPC分类号： C07K1/13 ,  C07H19/16 ,  C12N9/84 ,  A01K67/027 ,  C12N15/85

CPC分类号： C07K1/13 ,  A01K67/0275 ,  A01K67/0278 ,  A01K2217/206 ,  A01K2227/105 ,  A01K2267/0362 ,  A01K2267/0393 ,  C07H19/16 ,  C12N9/84 ,  C12N15/8509 ,  C12Y305/01011

摘要： Disclosed herein are compositions comprising blocked puromycin analogs that are converted into active puromycin analogs upon the activity of a penicillin acylase. Also disclosed are methods of using blocked puromycin analogs to label proteins in a selected cell type in vivo in a transgenic multicellular organism that expresses a penicillin acylase within the selected cell type. Also disclosed are transgenic mice expressing a penicillin acylase within a selected cell type.

摘要翻译： 本文公开了包含封闭的嘌呤霉素类似物的组合物，其在青霉素酰基转移酶的活性上转化为活性嘌呤霉素类似物。 还公开了使用封闭的嘌呤霉素类似物在所选择的细胞类型中表达青霉素酰基转移酶的转基因多细胞生物体内在体内选择的细胞类型中标记蛋白质的方法。 还公开了在所选细胞类型内表达青霉素酰基转移酶的转基因小鼠。

公开(公告)号：US09309537B2

公开(公告)日：2016-04-12

申请号：US13513100

申请日：2010-11-30

申请人： Takahiro Ochiya ,  Masaki Kawamata

发明人： Takahiro Ochiya ,  Masaki Kawamata

IPC分类号： C12N15/00 ,  C12N15/873 ,  A01K67/027 ,  C12N9/22 ,  C12N15/85 ,  C12N5/0735

CPC分类号： C12N15/873 ,  A01K67/0271 ,  A01K67/0276 ,  A01K2217/00 ,  A01K2217/052 ,  A01K2227/105 ,  A01K2267/0393 ,  C07K2319/81 ,  C12N5/0606 ,  C12N9/22 ,  C12N15/85 ,  C12N15/8509 ,  C12N2500/30 ,  C12N2501/065 ,  C12N2501/15 ,  C12N2501/998 ,  C12N2501/999

摘要： The present invention provides a preparation method of a chimeric embryo and a chimeric rat, which is characterized by contacting a rat pluripotent stem cell and a host embryo in the presence of an ES cell differentiation inhibitor. The method includes (a) a step for contacting a fertilized host embryo collected from a female rat and a rat pluripotent stem cell in the presence of an ES cell differentiation suppressant, and (b) a step for culturing the host embryo in contact with the rat pluripotent stem cell to form a chimeric embryo.

摘要翻译： 本发明提供嵌合胚胎和嵌合大鼠的制备方法，其特征在于在ES细胞分化抑制剂存在下使大鼠多能干细胞与宿主胚胎接触。 该方法包括：（a）在ES细胞分化抑制剂存在下，将从雌性大鼠和大鼠多能干细胞收集的受精宿主胚胎接触的步骤，和（b）与所述宿主胚胎接触的步骤 大鼠多能干细胞形成嵌合胚胎。

公开(公告)号：US20160066550A1

公开(公告)日：2016-03-10

申请号：US14801589

申请日：2015-07-16

申请人： SANOFI

发明人： James CAO ,  Karen CHANDROSS ,  Kyriakos D. ECONOMIDES ,  Harry Gregory POLITES ,  Daniel WEINSTOCK ,  Xiaoyou YING

IPC分类号： A01K67/027

CPC分类号： A01K67/0275 ,  A01K2217/052 ,  A01K2217/206 ,  A01K2227/105 ,  A01K2267/03 ,  A01K2267/0393 ,  C12N15/8509

摘要： A Myelin Basic Protein-luciferase bioimaging noninvasive model to visualize and quantify demyelination and remyelination events in the CNS at transcriptional level in vivo is provided. Luciferase-expressing transgenic animals were generated with myelin basic protein (MBP) promoter coupled to firefly luciferase reporter. The MBP-luci bioimaging model provides a means to monitor myelination status and the efficacy of a remyelination modulating test compound. An advantage of bioimaging is that a subject in a longitudinal study can serve as its own control. The same subject can be tracked over a demyelination and remyelination process continuously over a period of at least 10 weeks. This model enables normalization of individual animal imaging response and provides quality data with considerably reduced variance. In addition, because cohorts of animals need not be sacrificed at different time points, reduction in the number necessary for a compound efficacy study is possible.

公开(公告)号：US20160002302A1

公开(公告)日：2016-01-07

申请号：US14862926

申请日：2015-09-23

申请人： The Board of Trustees of the Leland Stanford Junior University

发明人： Karl Deisseroth ,  Ofer Yizhar ,  Lief Fenno ,  Peter Hegemann ,  Matthias Prigge

IPC分类号： C07K14/405 ,  G01N33/50 ,  A61N5/06 ,  C12N13/00

CPC分类号： C07K14/405 ,  A01K67/0275 ,  A01K2217/052 ,  A01K2227/105 ,  A01K2267/0393 ,  A61K38/00 ,  A61N5/06 ,  A61N2005/0663 ,  C07K19/00 ,  C07K2319/00 ,  C07K2319/60 ,  C12N13/00 ,  G01N33/5058 ,  G01N2500/10

摘要： Provided herein are compositions comprising light-activated chimeric proteins expressed on plasma membranes and methods of using the same to selectively depolarize excitatory or inhibitory neurons.

公开(公告)号：US20150368666A1

公开(公告)日：2015-12-24

申请号：US14765665

申请日：2014-02-03

申请人： ETH Zurich

发明人： Katrin Rössger ,  Martin Fussenegger

IPC分类号： C12N15/85 ,  A61K35/12

CPC分类号： C12N15/85 ,  A01K2207/12 ,  A01K2227/105 ,  A01K2267/0393 ,  A61K9/0024 ,  A61K9/5036 ,  A61K35/12 ,  A61K38/10 ,  A61K38/105 ,  A61K38/1825 ,  A61K38/1866 ,  A61K38/22 ,  A61K38/2207 ,  A61K38/2264 ,  A61K38/26 ,  A61K38/27 ,  A61K47/6901 ,  C12N15/09 ,  C12N2800/107 ,  C12N2800/60 ,  C12N2830/002

摘要： The invention relates to vectors and mammalian cells in a system useful for switching on or switching off gene expression in response to fatty acids, and a method of treating diet-induced obesity. In particular, a synthetic intracellular lipid-sensing receptor was constructed that constantly monitors blood fatty acid levels, processes diet-associated hyperlipidemia and coordinates reversible and adjustable expression of the anorectic peptide hormone pramlintide to reduce dietary intake, blood fat levels and body weight.

摘要翻译： 本发明涉及可用于开启或关闭响应于脂肪酸的基因表达的系统中的载体和哺乳动物细胞，以及治疗饮食诱导的肥胖症的方法。 特别地，构建了合成的细胞内脂质感应受体，其不断地监测血液脂肪酸水平，处理饮食相关的高脂血症，并且协调厌食肽激素普兰林素的可逆和可调节的表达，以减少饮食摄入，血脂水平和体重。