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1. 发明申请

WO2017075261A1 ENGINEERING OF HUMANIZED CAR T-CELLS AND PLATELETS BY GENETIC COMPLEMENTATION 审中-公开
标题翻译：基因遗传学研究人源化汽车T细胞和血小板的工程化
公开(公告)号：WO2017075261A1
公开(公告)日：2017-05-04
申请号：PCT/US2016/059191
申请日：2016-10-27
申请人： RECOMBINETICS, INC.
发明人： FAHRENKRUG, Scott, C. , COOPER, Laurence , HACKETT, Perry, B. , CARLSON, Daniel, F.
IPC分类号： A01K67/027 , C12N15/09 , C12N5/10 , C12N5/12
CPC分类号： C12N15/8778 , A01K67/027 , A01K67/0271 , A01K2207/12 , A01K2217/075 , A01K2217/15 , A01K2227/108 , A01K2267/025 , C12N5/0636 , C12N5/0644 , C12N2510/00
摘要： Human or humanized tissues and organs suitable for transplant are disclosed herein. Gene editing of a host animal to knock out or disrupt genes responsible for the growth and/or differentiation of a target organ provides a niche for complementation of the missing genetic information by injecting that animal at an embryo stage with donor stem cells to complement the missing genetic information for the growth and development of the organ. The result is a chimeric animal in which the complemented tissue (human/humanized organ) matches the genotype and phenotype of the donor. Such organs may be made in a single generation and the stem cell may be taken or generated from the patient's own body. Multiple genes can be targeted for editing using targeted nucleases and homology directed repair (HOR) templates in vertebrate cells or embryos.
摘要翻译：本文公开了适合移植的人或人源化组织和器官。对宿主动物进行基因编辑以敲除或破坏负责靶器官生长和/或分化的基因，通过在供体干细胞的胚胎期注射该动物以补充缺失的遗传信息来补充缺失的遗传信息遗传信息用于器官的生长和发育。其结果是一种嵌合动物，其中补充的组织（人/人源化器官）与供体的基因型和表型相匹配。这样的器官可以制造一代，并且干细胞可以从患者自身身体采集或产生。可以使用靶向核酸酶和脊椎动物细胞或胚胎中的同源定向修复（HOR）模板对多个基因进行编辑。

2. 发明申请

WO2014144719A2 ANIMAL MODELS OF ATAXIA-TELANGIECTASIS (A-T) 审中-公开
标题翻译： ATAXIA-TELANGIECTASIS动物模型（A-T）
公开(公告)号：WO2014144719A2
公开(公告)日：2014-09-18
申请号：PCT/US2014/029248
申请日：2014-03-14
申请人： EXEMPLAR GENETICS, LLC
发明人： ROGERS, Christopher, S. , SWART, John, R.
IPC分类号： A01K67/027
CPC分类号： A01K67/0276 , A01K2217/075 , A01K2227/108 , A01K2267/0318 , C12N15/8778 , C12N2750/14143
摘要： The present invention provides transgenic, large non-human animal models of Ataxia-Telangiectasia, as well as methods of using such animal models in the identification and characterization of therapies for Ataxia-Telangiectasia.
摘要翻译：本发明提供了转铁蛋白 - 毛细血管扩张症的大型非人动物动物模型，以及使用这些动物模型鉴定和表征治疗共济失调 - 毛细血管扩张症的方法。

3. 发明申请

WO2014070887A1 CONTROL OF SEXUAL MATURATION IN ANIMALS 审中-公开
标题翻译：动物性欲成熟的控制
公开(公告)号：WO2014070887A1
公开(公告)日：2014-05-08
申请号：PCT/US2013/067502
申请日：2013-10-30
申请人： RECOMBINETICS, INC.
发明人： CARLSON, Daniel F. , FAHRENKRUG, Scott C. , LAUTH, Xavier
IPC分类号： C12N5/10
CPC分类号： A01K67/0276 , A01K2217/075 , A01K2227/101 , A01K2227/108 , A01K2227/40 , A01K2267/02 , C12N15/8778 , C12N15/907
摘要： A genetically modified livestock animal comprising a genome that comprises inactivation of a neuroendocrine gene selective for sexual maturation, with the inactivation of the gene preventing the animal from becoming sexually mature. Methods of using, and processes of making, the animals are taught. Conventional livestock raising practices focus on the role of sexual maturity in livestock in terms of optimizing breeding and parturition.
摘要翻译：一种转基因家禽动物，其包含基因组，所述基因组包括使对成熟有选择性的神经内分泌基因的灭活，所述基因的失活阻止所述动物变得性成熟。教授动物的使用方法和制作过程。传统的养殖做法侧重于在优化育种和分娩方面，在家畜中性成熟的作用。

4. 发明申请

WO2014066505A1 DOUBLE KNOCKOUT (GT/CMAH-KO) PIGS, ORGANS AND TISSUES 审中-公开
标题翻译：双重堵塞（GT / CMAH-KO）皮，器官和组织
公开(公告)号：WO2014066505A1
公开(公告)日：2014-05-01
申请号：PCT/US2013/066387
申请日：2013-10-23
申请人： INDIANA UNIVERSITY RESEARCH & TECHNOLOGY CORPORATION , TECTOR, A., Joseph
发明人： TECTOR, A., Joseph
IPC分类号： A01K67/027 , C12N9/02 , C12N9/22 , C12N9/40 , C12N15/877
CPC分类号： A01K67/0276 , A01K2217/00 , A01K2217/075 , A01K2217/15 , A01K2227/108 , A01K2267/025 , A61K35/407 , C07K2319/80 , C12N9/0071 , C12N9/1048 , C12N9/22 , C12N9/2465 , C12N15/8509 , C12N15/8778 , C12N15/907
摘要： The invention provides double knockout transgenic pigs (GT/CMAH-KO pigs) lacking expression of any functional aGAL and CMAH. Double knockout GT/CMAH-KO transgenic organs, tissues and cells are also provided. Methods of making and using the GT/CMAH-KO pigs and tissue are also provided.
摘要翻译：本发明提供缺乏表达任何功能性aGAL和CMAH的双重敲除转基因猪（GT / CMAH-KO猪）。还提供了双重敲除GT / CMAH-KO转基因器官，组织和细胞。还提供了制造和使用GT / CMAH-KO猪和组织的方法。

5. 发明申请

WO2013192316A1 GENETICALLY MODIFIED ANIMALS AND METHODS FOR MAKING THE SAME 审中-公开
标题翻译：遗传改良动物及其制备方法
公开(公告)号：WO2013192316A1
公开(公告)日：2013-12-27
申请号：PCT/US2013/046590
申请日：2013-06-19
申请人： RECOMBINETICS, INC.
发明人： FAHRENKRUG, Scott, C. , CARLSON, Daniel, F.
IPC分类号： A01K67/027 , C12N15/63 , C12N15/85 , C12N5/073
CPC分类号： C12N15/907 , A01K67/0275 , A01K67/0276 , A01K2207/05 , A01K2217/072 , A01K2217/075 , A01K2217/15 , A01K2227/101 , A01K2227/108 , A01K2227/30 , A01K2267/02 , A01K2267/03 , C12N9/22 , C12N15/8509 , C12N15/8771 , C12N15/8778 , C12N2750/14143 , C12N2800/24 , C12N2999/007
摘要： Compositions and methods for use of TALENs to make genetically modified livestock or other animals are set forth. Some of the embodiments of the invention provide for making an founder animal that is completely free of all unplanned genetic modifications. Some embodiments are directed to removing genetic faults in established breeds without making other alterations to the genome. Other embodiments are directed to particular tools or processes such as a TALENs with a preferred truncation.
摘要翻译：阐述了使用TALEN制作转基因家禽或其他动物的组合物和方法。本发明的一些实施方案提供了制造完全没有所有非计划遗传修饰的创始动物。一些实施方案涉及去除已确定品种的遗传缺陷而不对基因组进行其它改变。其他实施方案涉及特定的工具或过程，例如具有优选截断的TALEN。

6. 发明申请

WO2008106986A1 PIG MODEL FOR PSORIASIS 审中-公开
标题翻译： PSORIASIS的PIG模型
公开(公告)号：WO2008106986A1
公开(公告)日：2008-09-12
申请号：PCT/DK2008/050059
申请日：2008-03-07
申请人： AARHUS UNIVERSITET , LEO PHARMA A/S , Syddansk Universitet , KRAGH, Peter Michael , BOLUND, Lars Axel , KRISTIANSEN, Karsten , SØRENSEN, Charlotte Brandt , MIKKELSEN, Jacob Giehm , STAUNSTRUP, Nicklas Heine , PETERSEN, Thomas Kongstad , SVENSSON, Lars
发明人： KRAGH, Peter Michael , BOLUND, Lars Axel , KRISTIANSEN, Karsten , SØRENSEN, Charlotte Brandt , MIKKELSEN, Jacob Giehm , STAUNSTRUP, Nicklas Heine , PETERSEN, Thomas Kongstad , SVENSSON, Lars
IPC分类号： C12N15/87 , A01K67/027 , C12N5/10 , G01N33/50
CPC分类号： A01K67/0273 , A01K2207/15 , A01K2217/00 , A01K2227/108 , A01K2267/0325 , C12N15/8778 , C12N2517/04 , C12N2800/30 , C12N2800/90 , G01N2500/00
摘要： The present invention relates to a genetically modified pig as a model for studying psoriasis. The modified pig model displays one or more phenotypes associated with psoriasis. Disclosed is also a modified pig comprising a mutation in the endogenous ILK-I Ra, JunB/cJun, CD18, IKK2, and/or LIG1 gene, and/or a human, porcine and/or murine PPARs, PPAR-δ, lκB-α, STAT3c, lntegrin beta 1, lntegrin alpha 2, MEK1, Amphiregulin, BMP-6, VEGF, JunBΔec-JunΔep, IL-I a, TGF.beta 1, CD18 hypo, Cre- IIKK2 fl7fl, Dsg1, SCCE, TGF-a, TNF-a, IL-20, IFN-g, LIG1 KO, KGF, IL-6, PAFR1 Cre/lkk2FL/FL, IL1 R, Dsg3, IFN-gamma, p40, ILI Ra, IKK2, JunB/c-Jun, and/or LIG1 gene, transcriptional and/or translational product or part thereof. The invention further relates to methods for producing the modified pig; and methods for evaluating the effect of a therapeutical treatment of psoriasis, for screening the efficacy of a pharmaceutical composition, and a method for treatment of human being suffering from psoriasis are disclosed.
摘要翻译：本发明涉及一种转基因猪作为研究牛皮癣的模型。修改后的猪模型显示与牛皮癣相关的一种或多种表型。还公开了包含内源性ILK-IRa，JunB / cJun，CD18，IKK2和/或LIG1基因和/或人，猪和/或鼠PPARs，PPAR-d， Ba，STAT3c，组蛋白β1，整合素α2，MEK1，双调蛋白，BMP-6，VEGF，JunB？ec-Jun？ep，IL-1α，TGFβ1，CD18 hypo，Cre-IIKK2 fl7fl，Dsg1， SCC，TGF-α，TNF-α，IL-20，IFN-g，LIG1KO，KGF，IL-6，PAFR1 Cre / lkk2FL / FL，IL1R，Dsg3，IFN-γ，p40，ILI Ra，IKK2， JunB / c-Jun和/或LIG1基因，转录和/或翻译产物或其一部分。本发明还涉及生产改性猪的方法; 公开了用于评价银屑病治疗效果，用于筛选药物组合物的功效以及治疗患有牛皮癣的方法的方法。

7. 发明申请

WO2005032245A1 METHOD OF PRODUCING TRANSGENIC PIGS EXPRESSING GREEN FLUORESCENT PROTEIN 审中-公开
标题翻译：生产表达绿色荧光蛋白的转基因小鼠的方法
公开(公告)号：WO2005032245A1
公开(公告)日：2005-04-14
申请号：PCT/KR2004/001414
申请日：2004-06-14
申请人： MGENBIO, INC. , PARK, Kwang-Wook , SEOL, Jae-Goo , HEO, Ki-Nam , LEE, Seung-Hyeon , HONG, Seung-Pyo , CHOI, Ki-Myung
发明人： PARK, Kwang-Wook , SEOL, Jae-Goo , HEO, Ki-Nam , LEE, Seung-Hyeon , HONG, Seung-Pyo , CHOI, Ki-Myung
IPC分类号： A01K67/027
CPC分类号： A01K67/0273 , A01K67/0275 , A01K2217/05 , A01K2227/108 , A01K2267/0393 , C12N15/8509 , C12N15/8778
摘要： The present invention relates to a method of producing a transgenic pig expressing green fluorescent protein (GFP). Particularly, the present invention relates to the method of producing the above transgenic pig including the steps of; (1) separating a somatic cell from a porcine fetus; (2) preparing the expression vector which includes a green fluorescent gene, followed by introducing the above expression vector into the above somatic cell; (3) selecting a cloned somatic cell transduced with the above expression vector and culturing that selected cell; (4) eliminating a nucleus from the oocyte of the donor pig, followed by fusing the resulting oocyte with the somatic cell to form a oocyte-somatic cell complex; and (5) transplanting the fused replicative oocyte into a surrogate pig and delivering piglets of present invention. The transgenic pigs expressing green fluorescent protein produced according to the method of the present invention can be effectively used as a transgenic animal model in the fields of the basic studies of science including cell therapy and in the production of a therapeutic protein by the transgenic animals.
摘要翻译：本发明涉及生产表达绿色荧光蛋白（GFP）的转基因猪的方法。特别地，本发明涉及生产上述转基因猪的方法，包括以下步骤：（1）从猪胎儿分离体细胞; （2）制备包含绿色荧光基因的表达载体，然后将上述表达载体导入上述体细胞; （3）选择用上述表达载体转导的克隆体细胞并培养所选择的细胞; （4）从供体猪的卵母细胞中消除细胞核，然后将所得卵母细胞与体细胞融合以形成卵母细胞体细胞复合体; 和（5）将融合的复制卵母细胞移植到替代猪中并递送本发明的仔猪。根据本发明的方法生产的表达绿色荧光蛋白的转基因猪可以有效地用作包括细胞治疗在内的科学基础研究领域和由转基因动物生产治疗性蛋白质的转基因动物模型。

8. 发明申请

WO2002072801A1 METHOD FOR THE PRODUCTION OF NUCLEAR TRANSFER EMBRYOS 审中-公开
标题翻译：生产核转移胚胎的方法
公开(公告)号：WO2002072801A1
公开(公告)日：2002-09-19
申请号：PCT/AU2002/000263
申请日：2002-03-08
申请人： GARELAG PTY LTD , RELAG PTY LTD , BOQUEST, Andrew, Craig , GRUPEN, Christopher, Gerald , NOTTLE, Mark, Brenton
发明人： BOQUEST, Andrew, Craig , GRUPEN, Christopher, Gerald , NOTTLE, Mark, Brenton
IPC分类号： C12N5/16
CPC分类号： C12N15/8778
摘要： Methods for the production of nuclear transfer embryos, nuclear transfer embryos and animals derived therefrom are described. The method generally comprises at least the steps of: providing at least one enucleated recipient cell; providing at least one donor cell or nucleus; providing a fusion media which is substantially free of calcium; placing said at least one enculeated recipient cell and at least one donor cell or nucleus in contact with one another to form couplets; and, fusing via electrofusion in said fusion media said at least one recipient cell with at least one donor cell or nucleus to form a nuclear transfer embryo.
摘要翻译：描述了从其衍生的核转移胚胎，核转移胚胎和动物的生产方法。该方法通常至少包括以下步骤：提供至少一个去核的受体细胞; 提供至少一个供体细胞或细胞核; 提供基本上不含钙的融合介质; 将所述至少一个包裹的受体细胞和至少一个供体细胞或细胞核相互接触以形成对联; 并且通过所述融合介质中的电融合将所述至少一个受体细胞与至少一个供体细胞或细胞核融合以形成核转移胚胎。

9. 发明申请

WO1995034636A1 UNGULATE EG CELL 审中-公开
标题翻译：甲壳虫细胞
公开(公告)号：WO1995034636A1
公开(公告)日：1995-12-21
申请号：PCT/JP1995001194
申请日：1995-06-15
申请人： MEIJI MILK PRODUCTS CO., LTD. , HASHIMOTO, Koichiro , MITANI, Tasuku , TAKAHASHI, Nobuko , NAKAO, Haruhiko , KAWASE, Eihachiro , MATSUI, Yasuhisa
发明人： MEIJI MILK PRODUCTS CO., LTD.
IPC分类号： C12N05/06
CPC分类号： C12N15/8778 , C12N5/0611
摘要： A porcine EG cell strain has been established which is derived from porcine primordial germ cells, has a multipotency or totipotency like that of an embryonic stem cell, and can be subcultured on feeder cells. The strain is reactive with a monoclonal antibody 4C9 prepared by using a mouse embryonic carcinoma cell F9 strain as the antigen. As the established EG cell strain can be readily subjected to gene manipulation under culture, in particular, gene targeting, it is easy to produce a variety of swine by gene manipulation.
摘要翻译：已经建立了源自猪原始生殖细胞的猪EG细胞株，具有如胚胎干细胞的多能性或全能性，并且可以在饲养细胞上传代培养。该菌株与通过使用小鼠胚胎癌细胞F9菌株作为抗原制备的单克隆抗体4C9反应。由于已建立的EG细胞株可以容易地在培养，特别是基因靶向下进行基因操作，因此通过基因操作容易地产生各种猪。

10. 发明申请

WO2018195129A1 EMBRYONIC CELL CULTURES AND METHODS OF USING THE SAME 审中-公开
公开(公告)号：WO2018195129A1
公开(公告)日：2018-10-25
申请号：PCT/US2018/028041
申请日：2018-04-17
申请人： UNIVERSITY OF MARYLAND, COLLEGE PARK
发明人： PARK, Chi-Hun , TELUGU, Bhanu Prakash V.L.
IPC分类号： C12N5/00 , C12N5/02 , C12N5/07 , C12N5/071 , C12N5/073 , C12N5/0735 , C12N5/075
CPC分类号： C12N5/0605 , A01K67/027 , A01K67/0275 , A01K2217/07 , A01K2227/108 , C12N15/8778 , C12N2510/00 , C12N2830/008
摘要： The invention provides a composition comprising an extraembryonic endodermal (XEN) cell and/or an embryonic fibroblast (EF) cell. The invention also provides a method of establishing a XEN cell line or a primary embryonic fibroblast (EF) cell line in vitro, the method comprising culturing a zygote or parthenote from a mammal for a time sufficient to produce one or more blastocysts; and culturing the one or more blastocysts on feeder cells in culture medium for a time sufficient to produce one or a plurality of XEN cells and/or one or a plurality of EF cells.

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