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31. 发明授权

US08551773B2 Methods and compositions relating to improved lentiviral vectors and their applications 有权
标题翻译：与改良的慢病毒载体及其应用相关的方法和组合物
公开(公告)号：US08551773B2
公开(公告)日：2013-10-08
申请号：US13622309
申请日：2012-09-18
申请人： Research Development Foundation
发明人： Didier Trono , Patrick Salmon
IPC分类号： C12N15/867 , C12N7/01
CPC分类号： A61K48/0058 , A61K31/7105 , A61K35/17 , A61K48/00 , C07K14/5434 , C07K14/55 , C07K14/70503 , C07K14/7051 , C07K14/755 , C07K16/00 , C07K16/46 , C07K2317/622 , C12N7/00 , C12N15/86 , C12N2740/15043 , C12N2740/15045 , C12N2740/16043 , C12N2740/16045 , C12N2740/16211 , C12N2800/108 , C12N2830/50 , C12N2840/20
摘要： The present invention provides HIV-derived lentivectors which are safe, highly efficient, and very potent for expressing transgenes for human gene therapy, especially, in human hematopoietic progenitor cells as well as in all other blood cell derivatives. The lentiviral vectors comprise a self-inactivating configuration for biosafety and promoters such as the EF1α promoter as one example. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element. These vectors therefore provide useful tools for genetic treatments such as inherited and acquired lympho-hematological disorders, gene-therapies for cancers especially the hematological cancers, as well as for the study of hematopoiesis via lentivector-mediated modification of human HSCs.
摘要翻译：本发明提供了用于人基因治疗特别是人类造血祖细胞以及所有其他血细胞衍生物中表达转基因的安全，高效和非常有效的HIV衍生的慢病毒药物。慢病毒载体包括生物安全性的自灭活构型和启动子如EF1α启动子作为一个实例。还描述了另外的启动子。载体还可以包含额外的转录增强元件，例如木吸虫肝炎病毒转录后调节元件。因此，这些载体为遗传治疗提供了有用的工具，例如遗传和获得性淋巴 - 血液学障碍，癌症特别是血液癌症的基因治疗，以及通过慢性介导的人类HSC修饰研究造血功能。

32. 发明申请

US20130024958A1 LENTIVIRAL VECTORS THAT PROVIDE IMPROVED EXPRESSION AND REDUCED VARIEGATION AFTER TRANSGENESIS 审中-公开
标题翻译：转基因后提供改进表达和减少变异的生殖细胞
公开(公告)号：US20130024958A1
公开(公告)日：2013-01-24
申请号：US13463214
申请日：2012-05-03
申请人： Patrick Stern , Stephen Kissler
发明人： Patrick Stern , Stephen Kissler
IPC分类号： C12N15/867 , A01K67/027 , C12N5/10
CPC分类号： C12N15/86 , A01K2217/075 , C12N2740/16043 , C12N2830/46
摘要： The present invention provides new lentiviral vectors that include an anti-repressor element (ARE) and, optionally, a scaffold attachment region (SAR). The lentiviral vectors provide expression of a heterologous nucleic acid in at least 50% of the cells of multiple cell types when used for lentiviral transgenesis. In certain embodiments of the invention the heterologous nucleic acid encodes an RNAi agent such as an shRNA. The invention further provides transgenic nonhuman animals generated using a lentiviral vector that includes an ARE and optional SAR. In addition, the invention provides a variety of methods for using the vectors including for achieving gene silencing in eukaryotic cells and transgenic animals, and methods of treating disease. The invention also provides animal models of human disease in which one or more genes is functionally silenced using a lentiviral vector of the invention.
摘要翻译：本发明提供了新的慢病毒载体，其包括抗阻遏物元件（ARE）和任选的支架附着区域（SAR）。当用于慢病毒转基因时，慢病毒载体在至少50％的多种细胞类型的细胞中提供异源核酸的表达。在本发明的某些实施方案中，异源核酸编码RNAi试剂例如shRNA。本发明还提供使用包含ARE和任选的SAR的慢病毒载体产生的转基因非人动物。此外，本发明提供了使用包括用于在真核细胞和转基因动物中实现基因沉默的载体的各种方法，以及治疗疾病的方法。本发明还提供使用本发明的慢病毒载体将一种或多种基因功能性沉默的人类疾病的动物模型。

33. 发明申请

US20130022989A1 DENTAL STEM CELL REPROGRAMMING 审中-公开
标题翻译：牙齿干细胞转录
公开(公告)号：US20130022989A1
公开(公告)日：2013-01-24
申请号：US13381063
申请日：2010-06-25
申请人： Jeremy J. Mao , Mo Chen
发明人： Jeremy J. Mao , Mo Chen
IPC分类号： C12N15/867 , C12Q1/04 , G01N33/566 , C12N5/10
CPC分类号： C12N5/0696 , C12N2501/11 , C12N2501/115 , C12N2501/602 , C12N2501/603 , C12N2501/605 , C12N2501/606 , C12N2501/608 , C12N2506/1361 , C12N2510/00 , C12N2799/027
摘要： Provided is dental stem cell comprising an Oct3/4 transgene. Also provided is a method of making a pluripotent stem cell. Additionally, a method of preparing an insulin-secreting cell is provided. Further provided is an insulin-secreting cell prepared by that method. A method of preparing a chondrocyte-like cell is also provided, as is a chondrocyte-like cell prepared by that method. Additionally provided is a method of preparing a myocyte-like cell. Also, a myocyte-like cell prepared by that method is provided. A method of preparing a hair follicle-like cell is additionally provided, as is a hair follicle-like cell prepared by that method. A method of preparing a neuron-like cell is additionally provided, as is a neuron-like cell prepared by that method.
摘要翻译：提供了包含Oct3 / 4转基因的牙干细胞。还提供了制备多能干细胞的方法。另外，提供了制备胰岛素分泌细胞的方法。进一步提供通过该方法制备的胰岛素分泌细胞。还提供了制备软骨细胞样细胞的方法，以及通过该方法制备的软骨细胞样细胞。另外提供了制备肌细胞样细胞的方法。此外，提供通过该方法制备的肌细胞样细胞。还提供了制备毛囊状细胞的方法，以及通过该方法制备的毛囊样细胞。另外提供了制备神经元样细胞的方法，以及通过该方法制备的神经元样细胞也是如此。

34. 发明申请

US20120308641A1 Compositions and Methods for Enhancing Coagulation Factor VIII Function 有权
标题翻译：增强凝血因子VIII功能的组合物和方法
公开(公告)号：US20120308641A1
公开(公告)日：2012-12-06
申请号：US13437486
申请日：2012-04-02
申请人： Valder Arruda , Rodney M. Camire , Nicholas Iacobelli
发明人： Valder Arruda , Rodney M. Camire , Nicholas Iacobelli
IPC分类号： C07K14/755 , A61K38/37 , C12N15/861 , A61P7/04 , C12N5/10 , C12N15/867 , C12N15/86 , C12N15/12 , A61K9/127
CPC分类号： C07K14/755 , A61K38/00
摘要： Factor VIII variants and methods of use thereof are disclosed.
摘要翻译：公开了VIII因子变体及其使用方法。

35. 发明申请

US20120294838A1 Regulation of endogenous gene expression in cells using zinc finger proteins 审中-公开
标题翻译：使用锌指蛋白调节细胞内源性基因表达
公开(公告)号：US20120294838A1
公开(公告)日：2012-11-22
申请号：US13068878
申请日：2011-05-23
申请人： George Norbert Cox III , Casey Christopher Case , Stephen P. Eisenberg , Eric Edward Jarvis , Sharon Kaye Spratt
发明人： George Norbert Cox III , Casey Christopher Case , Stephen P. Eisenberg , Eric Edward Jarvis , Sharon Kaye Spratt
IPC分类号： C12N15/86 , C12N15/867 , C12N15/861 , C12N15/864 , C12N15/869 , A61K35/12 , A61P35/00 , A61P31/10 , A61P33/02 , A61P31/04 , A61P31/12 , A61P9/10 , A61P9/00 , A61P19/02 , A61P37/00 , A61K48/00 , C12N5/071
CPC分类号： A01K67/0275 , A01K2217/05 , A61K48/00 , A61K48/005 , C07K14/47 , C07K14/52 , C07K2319/00 , C07K2319/71 , C07K2319/81 , C12N15/63 , C12N15/67 , C12N15/8216 , C12N15/85 , C12N2830/002 , C12N2830/005 , C12N2830/008 , C12N2830/85 , C12Q1/66 , C12Q1/6897 , G01N33/5091
摘要： The present invention provides methods for modulating expression of endogenous cellular genes using recombinant zinc finger proteins.
摘要翻译：本发明提供了使用重组锌指蛋白调节内源细胞基因表达的方法。

36. 发明申请

US20120237969A1 Method of Preparing Cells Susceptible to Transmissible Spongiform Encephalopathy and the Creation of TSE Persistently Infected Cells 有权
标题翻译：准备感染传染性海绵状脑病的细胞和TSE持续感染细胞的产生方法
公开(公告)号：US20120237969A1
公开(公告)日：2012-09-20
申请号：US13512010
申请日：2010-11-22
申请人： Dong-Seob Tark , Hyo-Jin Kim , Hyun-Joo Shon , Yoon-Hee Lee , Min-Jeong Kim , Eun-Im Yun , In-Soo Cho , Chang-Hee Kweon , Yi-Seok Joo , Otto Windl , Michael Neale
发明人： Dong-Seob Tark , Hyo-Jin Kim , Hyun-Joo Shon , Yoon-Hee Lee , Min-Jeong Kim , Eun-Im Yun , In-Soo Cho , Chang-Hee Kweon , Yi-Seok Joo , Otto Windl , Michael Neale
IPC分类号： C12N15/867 , C12Q1/04 , C12N5/10
CPC分类号： C12N7/00 , C12N2740/15043 , C12N2790/10051
摘要： Disclosed is a method for preparation of transmissible spongiform encephalopathy (TSE) susceptible cells, and such cells may be efficiently used in diagnosis of TSE, etiology studies and development of novel therapeutics, etc.
摘要翻译：本发明公开了传染性海绵状脑病（TSE）敏感细胞的制备方法，可有效地用于TSE诊断，病因学研究和新型治疗药物的开发等。

37. 发明申请

US20120213738A1 Cardiac Repair By Reprogramming of Cardiac Fibroblasts Into Cardiomyocytes 有权
标题翻译：通过心脏成纤维细胞重编程心肌细胞进行心脏修复
公开(公告)号：US20120213738A1
公开(公告)日：2012-08-23
申请号：US13402609
申请日：2012-02-22
申请人： Young-Jae Nam , Kunhua Song , Eric N. Olson
发明人： Young-Jae Nam , Kunhua Song , Eric N. Olson
IPC分类号： A61K48/00 , C12N15/85 , A61P9/10 , C12N15/861 , C12N15/867 , A61K38/17 , C12N5/071 , C12N15/86
CPC分类号： C12N5/0657 , A61K31/7105 , A61K38/00 , A61K38/17 , A61K45/06 , C07K14/4702 , C12N2501/40 , C12N2770/00043
摘要： The present invention involves the use of transcription factors including Tbx5, Mef2C, Hand2, myocardin and Gata4 to reprogram cardiac fibroblasts into cardiomyocytes, both in vitro and in vivo. Such methods find particular use in the treatment of patients post-myocardial infarction to prevent or limit scarring and to promote myocardial repair.
摘要翻译：本发明涉及使用包括Tbx5，Mef2C，Hand2，心肌素和Gata4在内的转录因子在体外和体内将心脏成纤维细胞重编程为心肌细胞。这种方法特别用于治疗心肌梗塞后的患者，以预防或限制瘢痕形成并促进心肌修复。

38. 发明申请

US20120183573A1 INTRACELLULAR VIRAL VECTOR DELIVERY METHOD EMPLOYING IRON ION/VIRAL VECTOR COMPOSITE 有权
标题翻译：使用铁离子/病毒载体复合材料的细胞内载体递送方法
公开(公告)号：US20120183573A1
公开(公告)日：2012-07-19
申请号：US13499101
申请日：2010-09-28
申请人： Young Chul Sung , Sang Hoon Park , Ki Seok Park
发明人： Young Chul Sung , Sang Hoon Park , Ki Seok Park
IPC分类号： A61K39/385 , C12N15/86 , C12N15/867 , C12N15/864 , C12N15/863 , A61P31/12 , C12N15/861
CPC分类号： C12N15/86 , C12N15/00 , C12N15/63 , C12N15/861 , C12N15/863 , C12N15/8636 , C12N15/864 , C12N15/867 , C12N2710/10343 , C12N2710/10345 , C12N2810/10
摘要： The present invention relates to an intracellular viral vector delivery method employing an iron ion/viral vector composite. The iron ion/viral vector composite according to the present invention is not dependent on the expression of CAR and so improves the efficiency of delivery of viral vectors and gene expression in cells of diverse types, and has an outstanding virus-neutralizing antibody escape performance, exhibits little cytotoxicity and is outstandingly stable even when subjected to iron ion processing at low concentration, and hence can be used to advantage in recombinant viral vaccine compositions.
摘要翻译：本发明涉及使用铁离子/病毒载体复合物的细胞内病毒载体递送方法。根据本发明的铁离子/病毒载体复合物不依赖于CAR的表达，因此提高病毒载体的传递效率和在不同类型的细胞中的基因表达，并且具有优异的病毒中和抗体逃逸性能，表现出很小的细胞毒性，即使在低浓度下进行铁离子处理也显着稳定，因此可用于重组病毒疫苗组合物中。

39. 发明申请

US20120122716A1 CELL LINE DERIVED FROM THE EPITHELIAL LINING OF THE HUMAN ENDOLYMPHATIC SAC IN THE INNER EAR 审中-公开
标题翻译：细胞系从人类内分泌系统的上皮细胞内皮细胞
公开(公告)号：US20120122716A1
公开(公告)日：2012-05-17
申请号：US13296136
申请日：2011-11-14
申请人： DAVID LIM , SUNG-KYUN MOON , HELGE RASK-ANDERSEN , JOHNG S. RHIM
发明人： DAVID LIM , SUNG-KYUN MOON , HELGE RASK-ANDERSEN , JOHNG S. RHIM
IPC分类号： C12Q1/68 , C12N15/86 , C12N15/867 , G01N27/62 , C12N5/10 , C12Q1/02 , C40B30/04 , C12N15/85 , C12N15/861
CPC分类号： G01N33/5044 , C12N5/0625 , C12N2510/04 , C12N2740/10041 , C12N2799/027 , G01N2800/14
摘要： In some embodiments, the invention relates to a stable, long-term human ES cell line. In other aspects, the invention relates to methods for establishing a stable long-term ES cell line and methods for screening therapeutic treatments for inner ear diseases, such as Meniere's disease.
摘要翻译：在一些实施方案中，本发明涉及稳定的长期人ES细胞系。在其他方面，本发明涉及建立稳定的长期ES细胞系的方法和用于筛选诸如梅尼埃病的内耳疾病的治疗方法。

40. 发明申请

US20120083033A1 PRODUCTION OF TRANSGENIC AVIANS USING IMPROVED RETROVIRAL VECTORS 有权
标题翻译：使用改进的逆转录矢量生产转基因生物
公开(公告)号：US20120083033A1
公开(公告)日：2012-04-05
申请号：US13179281
申请日：2011-07-08
申请人： ALEX J. HARVEY , JEFFREY C. RAPP
发明人： ALEX J. HARVEY , JEFFREY C. RAPP
IPC分类号： C12N5/10 , C12N15/867 , C12N15/11 , C12N15/85
CPC分类号： A01K67/0278 , A01K67/0275 , A01K2207/15 , A01K2217/052 , A01K2227/30 , A01K2267/01 , C07K14/56 , C07K14/70521 , C07K2319/30 , C12N7/00 , C12N15/8509 , C12N15/86 , C12N2015/8518 , C12N2740/11043 , C12N2800/24 , C12N2830/008 , C12N2830/15 , C12N2830/90
摘要： A transgenic avian containing in its genome an exogenous nucleotide sequence which includes a promoter component and a vector with reduced promoter interference wherein the exogenous nucleotide sequence is integrated into the genome and the avian.
摘要翻译：在其基因组中含有外源核苷酸序列的转基因禽类，其包含启动子组分和具有降低的启动子干扰的载体，其中外源核苷酸序列整合到基因组和禽类中。

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