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1. 发明申请

WO2019006127A1 SINGLE KIDNEY CELL-DERIVED ORGANOIDS 审中-公开
公开(公告)号：WO2019006127A1
公开(公告)日：2019-01-03
申请号：PCT/US2018/040022
申请日：2018-06-28
申请人： RUTGERS, THE STATE UNIVERSITY OF NEW JERSEY
发明人： SABAAWY, Hatem
IPC分类号： A61K9/00 , A61K35/12 , A61K35/22 , A61K35/44 , A61K49/10 , A61P13/12
CPC分类号： A61P13/12 , A61K35/22 , A61K49/0008 , C12N5/0686 , C12N2500/38 , C12N2501/105 , C12N2501/11 , C12N2501/165 , C12N2501/33 , C12N2501/39 , C12N2501/415 , C12N2501/727 , C12N2501/999 , C12N2513/00 , C12N2533/90
摘要： The present invention relates to organoids derived from a single cell, such as a kidney cancer cell, and methods and compositions relating to the production and use thereof, including cell culture medium for producing organoids and methods of personalized treatment for kidney cancer. The invention further provides a humanized mouse including a kidney organoid derived from a patient's kidney cell.

2. 发明申请

WO2018120610A1 一种Leg1蛋白、Leg1基因及其应用和药物审中-公开
公开(公告)号：WO2018120610A1
公开(公告)日：2018-07-05
申请号：PCT/CN2017/085350
申请日：2017-05-22
申请人：浙江大学
发明人：彭金荣 , 胡敏杰
IPC分类号： C07K14/47 , C12N15/12 , A61K38/17 , A61K45/00 , A61P3/10 , A61P3/04 , A61P3/00
CPC分类号： C07K14/4705 , A01K67/0275 , A01K67/0276 , A01K2217/072 , A01K2217/075 , A01K2227/105 , A01K2267/0362 , A61K38/00 , A61K38/17 , A61K49/0008 , A61P3/00 , A61P3/04 , A61P3/10 , C07K14/47 , C12N15/113 , C12N15/8509 , C12N2015/8527 , C12N2310/14
摘要：一种Leg1蛋白、Leg1基因及其应用和药物，是以mLeg1基因敲除小鼠为研究对象，利用遗传学，分子生物学，生物化学，细胞生物学的手段对mLeg1基因的功能进行了非常全面的研究，研究结果表明mLeg1蛋白可以通过EGFR调控体内Akt的信号，进而调控机体内的脂肪合成，该研究结果为后期通过人为干预hLeg1基因和hLeg1蛋白的表达水平来治疗人类肥胖症以及癌症病人化疗后的体质恢复提供了新的手段和思路。

3. 发明申请

WO2018065384A1 EXPRESSION PROFILES OF ABC AND SLC TRANSPORTERS IN HUMAN AND MINIPIG SKIN, LIVER AND KIDNEY 审中-公开
标题翻译： ABC和SLC转运蛋白在人和皮肤，肝和肾中的表达特征
公开(公告)号：WO2018065384A1
公开(公告)日：2018-04-12
申请号：PCT/EP2017/075032
申请日：2017-10-03
申请人： GALDERMA RESEARCH & DEVELOPMENT
发明人： OSMAN-PONCHET, Hanan
IPC分类号： C12Q1/68 , A61K49/00
CPC分类号： C12Q1/6886 , A01K2227/108 , A01K2267/03 , A61K49/0008 , C12Q1/6883 , C12Q2600/106 , C12Q2600/136 , C12Q2600/142
摘要： Novel methods and models involving minipig are discussed.
摘要翻译：
讨论了涉及小型猪的新方法和模型。

4. 发明申请

WO2017223457A1 GENERATION OF BROWN ADIPOSE TISSUE FROM PLURIPOTENT STEM CELLS IN VITRO 审中-公开
标题翻译：多潜能干细胞体外培养棕色脂肪组织的研究
公开(公告)号：WO2017223457A1
公开(公告)日：2017-12-28
申请号：PCT/US2017/039011
申请日：2017-06-23
申请人： THE BRIGHAM AND WOMEN'S HOSPITAL, INC.
发明人： POURQUIE, Olivier , CHAL, Jerome
IPC分类号： C12N5/07 , C12N5/071 , C12Q1/02 , C12Q1/68 , A61K35/35 , A61P5/00
CPC分类号： C12N5/0653 , A61K35/35 , A61K49/0008 , A61P5/00 , C12N2500/25 , C12N2501/105 , C12N2501/115 , C12N2501/12 , C12N2501/155 , C12N2501/385 , C12N2501/39 , C12N2501/395 , C12N2501/415 , C12N2506/02 , C12N2506/45 , C12N2533/54 , C12Q1/6881 , C12Q2600/158 , G01N33/5023 , G01N33/5041 , G01N33/5044 , G01N2800/044
摘要： A population of Brown adipose tissue (BAT) cells generated from embryonic stem cells (ES) or induced pluripotent stem cells (iPS), called iBAT, the use thereof, methods to obtain iBAT by directed differentiation of ES/iPS, and media compositions to obtain iBAT.
摘要翻译：从胚胎干细胞（ES）或诱导性多能干细胞（iPS）产生的称为iBAT的棕色脂肪组织（BAT）细胞群，其用途，通过定向分化获得iBAT的方法 ES / iPS和媒体组合来获得iBAT。

5. 发明申请

WO2017087780A1 NON-HUMAN ANIMALS HAVING A HUMANIZED LYMPHOCYTE-ACTIVATION GENE 3 审中-公开
标题翻译：具有人源化淋巴细胞活化基因的非人动物3
公开(公告)号：WO2017087780A1
公开(公告)日：2017-05-26
申请号：PCT/US2016/062733
申请日：2016-11-18
申请人： REGENERON PHARMACEUTICALS, INC.
发明人： MUJICA, Alexander O. , BUROVA, Elena , MURPHY, Andrew J.
IPC分类号： A01K67/027 , C07K14/705 , C07K14/47
CPC分类号： A01K67/0278 , A01K67/0276 , A01K2207/15 , A01K2217/072 , A01K2217/075 , A01K2217/15 , A01K2227/105 , A01K2267/01 , A01K2267/03 , A01K2267/0331 , A61K49/0008 , C07K14/70503
摘要： Non-human animals, and methods and compositions for making and using the same, are provided, wherein said non-human animals comprise a humanization of a Lymphocyte activation gene 3 (Lag3). Said non-human animals may be described, in some embodiments, as having a genetic modification to an endogenous Lag3 locus so that said non-human animals express a Lag3 polypeptide that includes a human portion and an endogenous portion (e.g., a non-human portion).
摘要翻译：提供了非人动物以及用于制备和使用它们的方法和组合物，其中所述非人动物包含淋巴细胞激活基因3（Lag3）的人源化。在一些实施方案中，所述非人动物可以描述为对内源性Lag3基因座具有遗传修饰，使得所述非人动物表达包含人部分和内源部分（例如非人类部分）。

6. 发明申请

WO2016205523A1 GENETICALLY MODIFIED NON-HUMAN ANIMALS AND METHODS RELATING TO COMPLEMENT DEPENDENT CYTOTOXICITY 审中-公开
标题翻译：遗传修饰的非人动物及相关依赖性细胞毒性相关方法
公开(公告)号：WO2016205523A1
公开(公告)日：2016-12-22
申请号：PCT/US2016/037882
申请日：2016-06-16
申请人： THE JACKSON LABORATORY , UNIVERSITY OF MASSACHUSETTS
发明人： SHULTZ, Leonard, D. , VERMA, Mohit, Kumar , GREINER, Dale, L. , BREHM, Michael, A.
IPC分类号： A01K67/027 , C07K14/705 , G01N33/15
CPC分类号： A01K67/0275 , A01K2217/07 , A01K2227/105 , A01K2267/0387 , A61K49/0008 , C07K14/472 , C12N15/907
摘要： The present invention relates generally to genetically modified non-human animals and immunodeficient non-human animals characterized by restored complement-dependent cytotoxicity, as well as methods and compositions for assessment of therapeutic antibodies in the genetically modified immunodeficient non- human animals. In specific aspects, the present invention relates to immunodeficient non-obese diabetic (NOD), A/J, A/He, AKR, DBA/2, NZB/BIN, B10.D2/oSn and other mouse strains genetically modified to restore complement-dependent cytotoxicity which is lacking in the unmodified immunodeficient mice. In further specific aspects, the present invention relates to NOD.Cg- Prkdc scid IL2rg tm1Wjl /SzJ (NSG), NOD. Cg- Rag1 tm1Mom IL 2rg tmlWjl /SzJ (NRG) and NOD.Cg- Prkdc scid IL2rg tm1Sug /JicTAc (NOG) mice genetically modified to restore complement-dependent cytotoxicity which is lacking in unmodified NSG, NRG and NOG mice. Methods for assessment of therapeutic antibodies or putative therapeutic antibodies in the genetically modified immunodeficient mice characterized by an intact complement system are provided according to specific aspects of the present invention.
摘要翻译：本发明一般涉及以遗传修饰的免疫缺陷型非人类动物评估治疗性抗体为基础的遗传修饰的非人类动物和以恢复的补体依赖性细胞毒性为特征的免疫缺陷型非人类动物，以及用于评估治疗性抗体的方法和组合物。在具体方面，本发明涉及免疫缺陷型非肥胖性糖尿病（NOD），A / J，A / He，AKR，DBA / 2，NZB / BIN，B10D2 / oSn和其他经遗传修饰以恢复补体的小鼠在未修饰的免疫缺陷小鼠中缺乏依赖性细胞毒性。在其它具体方面，本发明涉及NOD.Cg-Prkdc scid IL2rg tm1Wj1 / SzJ（NSG），NOD。 Cg-Rag1 tm1Mom IL2rg tmlWj1 / SzJ（NRG）和NOD.Cg-Prkdc scid IL2rg tm1Sug / JicTAc（NOG）小鼠遗传修饰以恢复未修饰的NSG，NRG和NOG小鼠缺乏的补体依赖性细胞毒性。根据本发明的具体方面提供了用完整的补体系统表征的遗传修饰的免疫缺陷小鼠中治疗性抗体或推定的治疗性抗体的评估方法。

7. 发明申请

WO2016187416A1 A HIGH-THROUGHPUT ASSAY FOR QUANTIFYING APPETITE AND DIGESTIVE DYNAMICS 审中-公开
标题翻译：用于量化猜测动力学的高精度测量
公开(公告)号：WO2016187416A1
公开(公告)日：2016-11-24
申请号：PCT/US2016/033252
申请日：2016-05-19
申请人： JORDI, Josua , ENGERT, Florian
发明人： JORDI, Josua , ENGERT, Florian
IPC分类号： G01N33/50 , G01N33/68 , A61K49/00
CPC分类号： G01N33/5088 , A01K2207/25 , A01K2227/40 , A61K49/0008 , A61K49/0032 , G01N33/6893 , G01N2800/02 , G01N2800/303
摘要： A high throughput method to determine compounds that alter appetite, satiation or digestion in vertebrates is provided. In one embodiment a method to assay a test compound in a teleost larvae or adult is disclosed, comprising: providing a plurality of teleost larvae or adults in wells of a multi-well substrate having a food source comprising an optically detectable, ingestible, lipophilic molecule and a test compound, wherein at least one well has an individual larvae or adult; simultaneously exposing the plurality of larvae or adults to a non- laser infrared light source; and simultaneously detecting or quantifying emissions from the molecule while the teleost larvae or adult is alive and freely moving. Also disclosed, in one embodiment, is a method to detect nutrient homeostasis in a zebrafish larvae or adult, comprising: providing a plurality of live zebrafish larvae or adults in individual wells of a multi-well substrate having a food source comprising an optically detectable, ingestable, lipohilic molecule; and detecting simultaneously with an infrared macroscope the presence and/or amount of the optically detectable, lipophilic molecule in the intestine of the live, freely moving zebrafish larvae or adult, thereby detecting or quantifying intake and/or digestion rates.
摘要翻译：提供了一种高通量的方法来确定在脊椎动物中改变食欲，饱食或消化的化合物。在一个实施方案中，公开了一种测定硬骨鱼幼虫或成年人中的测试化合物的方法，其包括：在多孔底物的孔中提供多个硬骨鱼幼虫或成虫，所述多孔底物具有包含可光学检测的可摄入的亲脂性分子和试验化合物，其中至少一个孔具有单独的幼虫或成虫; 同时将多个幼虫或成人暴露于非激光红外光源; 同时检测或量化来自分子的排放物，而硬骨幼虫或成年人是活的和自由移动的。在一个实施方案中还公开了一种检测斑马鱼幼虫或成年人中的营养物质稳态的方法，其包括：在多孔底物的单个孔中提供多个活斑马鱼幼虫或成虫，所述多孔底物具有包含光学可检测，可摄取的脂肪分子; 并且与红外显微镜同时检测活的，自由移动的斑马鱼幼虫或成年人的肠中的光学可检测的亲脂性分子的存在和/或量，从而检测或定量摄入和/或消化率。

8. 发明申请

WO2016168762A2 ANTI-PACAP ANTIBODIES AND USES THEREOF 审中-公开
标题翻译：抗PACAP抗体及其用途
公开(公告)号：WO2016168762A2
公开(公告)日：2016-10-20
申请号：PCT/US2016/027984
申请日：2016-04-15
申请人： ALDER BIOPHARMACEUTICALS, INC. , ALLISON, Daniel, S. , HENDRICKS, Lee , OJALA, Ethan Wayne , FAN, Pei , SMITH, Jeffrey, T.L. , LATHAM, John , KARASEK, Charlie , MULLIGAN, Jenny , SCALLEY-KIM, Michelle , STEWART, Erica , RUBIN, Vanessa Lisbeth , BILLGREN, Jens, J.
发明人： ALLISON, Daniel, S. , HENDRICKS, Lee , OJALA, Ethan Wayne , FAN, Pei , SMITH, Jeffrey, T.L. , LATHAM, John , KARASEK, Charlie , MULLIGAN, Jenny , SCALLEY-KIM, Michelle , STEWART, Erica , RUBIN, Vanessa Lisbeth , BILLGREN, Jens, J. , LOOMIS, Maria-Cristina , DUTZAR, Benjamin, H. , GARCIA-MARTINEZ, Leon
IPC分类号： C07K16/26 , A61K45/06
CPC分类号： C07K16/26 , A61K39/3955 , A61K39/39566 , A61K45/06 , A61K49/0004 , A61K49/0008 , A61K2039/505 , C07K16/4241 , C07K2317/24 , C07K2317/33 , C07K2317/34 , C07K2317/40 , C07K2317/76 , C07K2317/92 , G01N33/5088 , G01N33/74 , G01N2333/5757 , Y02A50/383 , Y02A50/51
摘要： The present invention is directed to antagonistic antibodies and antigen binding fragments thereof having binding specificity for PACAP. These antibodies inhibit, block or neutralize at least one biological effect associated with PACAP, e.g., vasodilation. In exemplary embodiments these antibodies and antigen binding fragments thereof may comprise specific VH, VL, and CDR polypeptides described herein. In some embodiments these antibodies and antigen binding fragments thereof bind to and/or compete for binding to specific epitope(s) on human PACAP. The invention is further directed to using these antagonistic anti-PACAP antibodies, and binding fragments thereof, for the diagnosis, assessment, and treatment of diseases and disorders associated with PACAP and conditions where antagonism of PACAP-related activities, such as vasodilation, mast cell degranulation, and/or neuronal activation, are therapeutically beneficial, e.g., headache and migraine indications.
摘要翻译：本发明涉及对PACAP具有结合特异性的拮抗性抗体及其抗原结合片段。这些抗体抑制，阻断或中和与PACAP相关的至少一种生物学作用，例如血管舒张。在示例性实施方案中，这些抗体及其抗原结合片段可以包含本文所述的特异性VH，VL和CDR多肽。在一些实施方案中，这些抗体及其抗原结合片段与人PACAP上的特异性表位结合和/或竞争结合特异性表位。本发明进一步涉及使用这些拮抗性抗PACAP抗体及其结合片段用于诊断，评估和治疗与PACAP相关的疾病和病症以及其中PACAP相关活性如血管舒张，肥大细胞的拮抗作用脱颗粒和/或神经元激活是治疗有益的，例如头痛和偏头痛适应症。

9. 发明申请

WO2016168760A1 USE OF ANTI-PACAP ANTIBODIES AND ANTIGEN BINDING FRAGMENTS THEREOF FOR TREATMENT, PREVENTION, OR INHIBITION OF PHOTOPHOBIA 审中-公开
标题翻译：使用抗PACAP抗体和其抗原结合片段治疗，预防或抑制PHOTOPHOBIA
公开(公告)号：WO2016168760A1
公开(公告)日：2016-10-20
申请号：PCT/US2016/027981
申请日：2016-04-15
申请人： ALDER BIOPHARMACEUTICALS, INC. , THE UNIVERSITY OF IOWA RESEARCH FOUNDATION , ALLISON, Daniel, S. , HENDRICKS, Lee , OJALA, Ethan Wayne , FAN, Pei , SMITH, Jeffrey, T.L. , LATHAM, John , KARASEK, Charlie , MULLIGAN, Jenny , SCALLEY-KIM, Michelle , STEWART, Erica , RUBIN, Vanessa Lisbeth , BILLGREN, Jens, J.
发明人： ALLISON, Daniel, S. , HENDRICKS, Lee , OJALA, Ethan Wayne , FAN, Pei , SMITH, Jeffrey, T.L. , LATHAM, John , KARASEK, Charlie , MULLIGAN, Jenny , SCALLEY-KIM, Michelle , STEWART, Erica , RUBIN, Vanessa Lisbeth , BILLGREN, Jens, J. , KUBURAS, Adisa , MASON, Bianca , SOWERS, Levi, P. , RUSSO, Andrew, F. , LOOMIS, Maria-Cristina , DUTZAR, Benjamin, H. , GARCIA-MARTINEZ, Leon
IPC分类号： A61P25/06 , A61K39/395
CPC分类号： C07K16/26 , A61K39/3955 , A61K39/39566 , A61K45/06 , A61K49/0004 , A61K49/0008 , A61K2039/505 , C07K16/4241 , C07K2317/24 , C07K2317/33 , C07K2317/34 , C07K2317/40 , C07K2317/76 , C07K2317/92 , G01N33/5088 , G01N33/74 , G01N2333/5757 , Y02A50/383 , Y02A50/51
摘要： This invention relates to methods of screening for anti-PACAP antibodies, or anti-PACAP receptor antibodies, and antigen binding fragments thereof, for potential use in treating or preventing PACAP-associated photophobia or light aversion, and therapeutic compositions containing and methods of using anti-PACAP antibodies, or anti-PACAP receptor antibodies, and antigen binding fragments thereof.
摘要翻译：本发明涉及筛选用于治疗或预防PACAP相关畏光症或轻度厌恶症的潜在用途的抗PACAP抗体或抗PACAP受体抗体及其抗原结合片段的方法，以及包含和使用抗PACAP抗体或抗PACAP受体抗体及其抗原结合片段的方法的治疗组合物。

10. 发明申请

WO2016095037A1 MODULATION AND DETECTION OF A NEURONAL ALTERNATIVE SPLICING REGULATORY NETWORK FOR TREATMENT AND DIAGNOSIS OF NEUROLOGICAL DISORDERS 审中-公开
标题翻译：调节和检测神经元替代性分离法规网络，用于治疗和诊断神经病
公开(公告)号：WO2016095037A1
公开(公告)日：2016-06-23
申请号：PCT/CA2015/051328
申请日：2015-12-15
申请人： BLENCOWE, Benjamin J. , QUESNEL-VALLIERES, Mathieu , IRIMIA, Manuel , RAJ, Bushra , CORDES, Sabine P.
发明人： BLENCOWE, Benjamin J. , QUESNEL-VALLIERES, Mathieu , IRIMIA, Manuel , RAJ, Bushra , CORDES, Sabine P.
IPC分类号： C12N5/071 , A01K67/027 , A61K31/7088 , A61K38/46 , A61K39/395 , A61P25/00 , A61P25/18 , C12N15/00 , C12N15/12 , C12N5/079 , C12N5/10 , C12Q1/00 , C12Q1/68 , G01N33/48 , G06F19/10
CPC分类号： A61K38/05 , A01K67/0276 , A01K67/0278 , A01K2217/052 , A01K2217/072 , A01K2217/077 , A01K2227/105 , A01K2267/0306 , A01K2267/0356 , A61K31/7088 , A61K31/713 , A61K38/46 , A61K45/06 , A61K49/0008 , C07K14/4702 , C12Q1/6883 , C12Q2600/156 , G01N33/5058 , G01N33/5088 , G01N33/6896 , G01N2800/28 , A61K2300/00
摘要： Methods for treatment and diagnosis of neurological disorders such as autism and autism spectrum disorder are disclosed. Also disclosed are modulators of alternative splicing regulators SRRM4 and/or SRRM3 for treating neurological disorders. Further disclosed are agents that modulate the expression of at least one splice variant for treating neurological disorders. Mouse models of neurological disorders having increased or decreased expression of SRRM4 and/or SRRM3 are also disclosed.
摘要翻译：公开了治疗和诊断自闭症和自闭症谱系障碍的神经障碍的方法。还公开了用于治疗神经障碍的可选剪接调节物SRRM4和/或SRRM3的调节剂。还公开了调节用于治疗神经障碍的至少一种剪接变体的表达的试剂。还公开了具有SRRM4和/或SRRM3的表达增加或减少的神经障碍的小鼠模型。

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