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1. 发明申请

WO2016196185A1 NON-HUMAN ANIMALS HAVING A DISRUPTION IN A C9ORF72 LOCUS 审中-公开
标题翻译：非人类动物在C9ORF72位置遭受破坏
公开(公告)号：WO2016196185A1
公开(公告)日：2016-12-08
申请号：PCT/US2016/034304
申请日：2016-05-26
申请人： REGENERON PHARMACEUTICALS, INC.
发明人： ATANASIO, Amanda , IKIZ, Burcin , GONG, Guochun , LACROIX-FRALISH, Michael L. , LAI, Ka-Man Venus , VALENZUELA, David M.
IPC分类号： A01K67/027 , C12N15/85 , C12N15/90
CPC分类号： A01K67/0276 , A01K67/02 , A01K2217/072 , A01K2217/075 , A01K2227/105 , A01K2267/0318 , A01K2267/0381 , A01K2267/0393 , A61K49/0008 , C12N15/8509 , C12N15/907
摘要： A non-human animal model for neurodegenerative and/or inflammatory diseases is provided, which non-human animal comprises a disruption in a C9ORF72 locus. In particular, non-human animals described herein comprise a deletion of an entire coding sequence of a C9ORF72 locus. Methods of identifying therapeutic candidates that may be used to prevent, delay or treat one or more neurodegenerative (e.g., amyotrophic lateral sclerosis (ALS, also referred to as Lou Gehrig's disease) and frontotemporal dementia (FTD)), autoimmune and/or inflammatory diseases (e.g., SLE, glomerulonephritis) are also provided.
摘要翻译：提供了用于神经变性和/或炎性疾病的非人动物模型，其非人类动物包括C9ORF72基因座中的破坏。特别地，本文所述的非人动物包含C9ORF72基因座的整个编码序列的缺失。识别可用于预防，延迟或治疗一种或多种神经变性（例如肌萎缩性侧索硬化（ALS，也称为Lou Gehrig氏病））和额颞痴呆（FTD））的自身免疫性和/或炎性疾病的治疗候选物的方法（例如，SLE，肾小球性肾炎）。

2. 发明申请

WO2016100857A1 STEM CELLS FOR MODELING TYPE 2 DIABETES 审中-公开
标题翻译：用于建模2型糖尿病的干细胞
公开(公告)号：WO2016100857A1
公开(公告)日：2016-06-23
申请号：PCT/US2015/066757
申请日：2015-12-18
申请人： REGENERON PHARMACEUTICALS, INC.
发明人： GONG, Guochun , LAI, Ka-Man Venus , VALENZUELA, David M.
IPC分类号： C12N5/074 , C12N15/10 , C12N15/113 , A61K35/39 , A61K35/12 , A61K35/545 , C12N5/071 , C12N15/63 , C12N15/90
CPC分类号： C12N5/0696 , A01K67/0271 , A01K2207/12 , A01K2227/105 , A01K2267/0362 , C12N5/0676 , C12N5/0678 , C12N15/111 , C12N15/90 , C12N2310/20 , C12N2501/119 , C12N2501/16 , C12N2506/45 , G01N33/507 , G01N33/5088 , G01N33/74 , G01N2333/62
摘要： The invention provides stem cell derived beta-pancreatic cells and animal models of T2D in which cells have been grafted. The stem cells bear a mutated form of SLC30A8 conferring protection or susceptibility to T2D. The cells and animal models can be used for drug screening as well as to provide insights into the mechanism of T2D and potentially new therapeutic and diagnostic targets.
摘要翻译：本发明提供了干细胞衍生的β-胰腺细胞和其中细胞被移植的T2D的动物模型。干细胞具有突变形式的SLC30A8，赋予对T2D的保护或易感性。细胞和动物模型可用于药物筛选，并提供对T2D的机制和潜在的新的治疗和诊断目标的见解。

3. 发明申请

WO2014130706A1 GENETIC MODIFICATION OF RATS 审中-公开
标题翻译： RATS的遗传修饰
公开(公告)号：WO2014130706A1
公开(公告)日：2014-08-28
申请号：PCT/US2014/017452
申请日：2014-02-20
申请人： REGENERON PHARMACEUTICALS, INC.
发明人： LEE, Jeffrey, D. , AUERBACH, Wojtek , HESLIN, David , FRENDEWEY, David , LAI, Ka-man Venus , VALENZUELA, David M.
IPC分类号： C12N5/07 , C12N5/10 , C12N5/00 , C12N5/02
CPC分类号： C12N15/8509 , A01K67/02 , A01K67/0271 , A01K67/0275 , A01K2207/12 , A01K2217/15 , A01K2227/105 , A61D19/04 , C12N5/0606 , C12N15/8775 , C12N2501/235 , C12N2501/727 , C12N2501/999 , C12N2510/00
摘要： Compositions and methods are provided for making rat pluripotent and totipotent cells, including rat embryonic stem (ES) cells. Compositions and methods for improving efficiency or frequency of germline transmission of genetic modifications in rats are provided. Such methods and compositions comprise an in vitro culture comprising a feeder cell layer and a population of rat ES cells or a rat ES cell line, wherein the in vitro culture conditions maintain pluripotency of the ES cell and comprises a media having mouse leukemia inhibitor factor (LIF) or an active variant or fragment thereof. Various methods of establishing such rat ES cell lines are further provided. Methods of selecting genetically modified rat ES cells are also provided, along with various methods to generate a transgenic rat from the genetically modified rat ES cells provided herein. Various kits and articles of manufacture are further provided.
摘要翻译：提供组合物和方法用于制备大鼠多能全细胞，包括大鼠胚胎干（ES）细胞。提供了用于提高大鼠遗传修饰的种系传播效率或频率的组合物和方法。这些方法和组合物包括体外培养物，其包含饲养细胞层和大鼠ES细胞群或大鼠ES细胞系，其中体外培养条件保持ES细胞的多能性，并且包含具有小鼠白血病抑制因子（ LIF）或其活性变体或片段。进一步提供了建立这种大鼠ES细胞系的各种方法。还提供了选择遗传修饰的大鼠ES细胞的方法，以及从本文提供的遗传修饰的大鼠ES细胞产生转基因大鼠的各种方法。还提供了各种套件和制品。

4. 发明申请

WO2015188109A1 METHODS AND COMPOSITIONS FOR MODIFYING A TARGETED LOCUS 审中-公开
标题翻译：用于修改目标位置的方法和组合
公开(公告)号：WO2015188109A1
公开(公告)日：2015-12-10
申请号：PCT/US2015/034503
申请日：2015-06-05
申请人： REGENERON PHARMACEUTICALS, INC.
发明人： AUERBACH, Wojtek , FRENDEWEY, David , DROGUETT, Gustavo , GAGLIARDI, Anthony , KUNO, Junko , VALENZUELA, David M.
IPC分类号： C12N15/90
CPC分类号： C12N15/907 , A01K67/0278 , A01K2217/072 , A01K2227/105 , A01K2267/0387 , C07K14/7051 , C12N15/64 , C12N2810/00
摘要： Methods and compositions are provided for modifying one or more target loci in a cell. Such methods comprise providing a cell comprising a first polynucleotide encoding a first selection marker operably linked to a first promoter active in the cell, wherein the first polynucleotide further comprises a first recognition site for a first nuclease agent. A first nuclease agent is introduced into a cell, wherein the first nuclease agent induces a nick or double-strand break at the first recognition site. Further introduced into the cell is a first targeting vector comprising a first insert polynucleotide flanked by a first and a second homology arm that correspond to a first and a second target site located in sufficient proximity to the first recognition site. At least one cell is then identified comprising in its genome the first insert polynucleotide integrated at the target locus.
摘要翻译：提供了用于修饰细胞中一个或多个靶基因座的方法和组合物。这样的方法包括提供包含编码第一选择标记的第一多核苷酸的细胞，所述第一选择标记可操作地连接到细胞中活性的第一启动子，其中所述第一多核苷酸还包含第一核酸酶试剂的第一识别位点。将第一核酸酶试剂引入细胞中，其中第一核酸酶试剂在第一识别位点诱导切口或双链断裂。进一步引入细胞中的是第一靶向载体，其包含侧翼于第一和第二同源性臂的第一插入核苷酸，所述第一和第二同源性臂对应于位于第一识别位点附近的第一和第二靶位点。然后鉴定至少一个细胞，其在其基因组中包含整合在靶标位点的第一插入多核苷酸。

5. 发明申请

WO2014172489A2 TARGETED MODIFICATION OF RAT GENOME 审中-公开
标题翻译：大鼠基因组的靶向修饰
公开(公告)号：WO2014172489A2
公开(公告)日：2014-10-23
申请号：PCT/US2014/034412
申请日：2014-04-16
申请人： REGENERON PHARMACEUTICALS, INC.
发明人： LEE, Jeffrey D. , MUJICA, Alexander, O. , AUERBACH, Wojtek , LAI, Ka-man Venus , VALENZUELA, David M. , YANCOPOULOS, George D.
IPC分类号： A01K67/027 , C12N15/85
CPC分类号： C12N15/8509 , A01K67/0276 , A01K67/0278 , A01K2217/07 , A01K2227/105 , A01K2267/0362 , A01K2267/0381 , A61D19/04 , C07K14/7155 , C07K14/775 , C12N15/907 , C12N2015/8527 , C12N2800/30 , C12N2810/00
摘要： Compositions and methods are provided for modifying a rat genomic locus of interest using a large targeting vector (LTVEC) comprising various endogenous or exogenous nucleic acid sequences as described herein. Compositions and methods for generating a genetically modified rat comprising one or more targeted genetic modifications in their germline are also provided. Compositions and methods are provided which comprise a genetically modified rat or rat cell comprising a targeted genetic modification in the rat interleukin-2 receptor gamma locus, the rat ApoE locus, the rat Rag2 locus, the rat Rag1 locus and/or the rat Rag2/Rag1 locus. The various methods and compositions provided herein allows for these modified loci to be transmitted through the germline.
摘要翻译：提供了使用包含如本文所述的各种内源或外源核酸序列的大靶向载体（LTVEC）修饰感兴趣的大鼠基因组基因座的组合物和方法。还提供了用于产生在其种系中包含一种或多种靶向遗传修饰的遗传修饰的大鼠的组合物和方法。提供了组合物和方法，其包含在大鼠白细胞介素-2受体γ基因座，大鼠ApoE基因座，大鼠Rag2基因座，大鼠Rag1基因座和/或大鼠Rag2基因座中包含靶向遗传修饰的基因修饰的大鼠或大鼠细胞。 Rag1基因座。本文提供的各种方法和组合物允许这些修饰的基因座通过种系传播。

6. 发明申请

WO2016081923A2 METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATION USING PAIRED GUIDE RNAs 审中-公开
标题翻译：使用配对指南RNA对目标遗传修饰的方法和组合物
公开(公告)号：WO2016081923A2
公开(公告)日：2016-05-26
申请号：PCT/US2015/062023
申请日：2015-11-20
申请人： REGENERON PHARMACEUTICALS, INC. , MURPHY, Andrew J.
发明人： MURPHY, Andrew J. , FRENDEWEY, David , LAI, Ka-Man Venus , AUERBACH, Wojtek , LEE, Jeffrey D. , MUJICA, Alexander O. , DROGUETT, Gustavo , TRZASKA, Sean , HUNT, Charleen , GAGLIARDI, Anthony , VALENZUELA, David M. , VORONINA, Vera , MACDONALD, Lynn , YANCOPOULOS, George D.
IPC分类号： C12N15/90
CPC分类号： C12N15/907 , A01K67/0275 , A01K2227/105 , C12N9/22 , C12N15/102 , C12N15/8509 , C12Q1/6888
摘要： Compositions and methods are provided for creating and promoting biallelic targeted modifications to genomes within cells and for producing non-human animals comprising the modified genomes. Also provided are compositions and methods for modifying a genome within a cell that is heterozygous for an allele to become homozygous for that allele. The methods make use of Cas proteins and two or more guide RNAs that target different locations within the same genomic target locus. Also provided are methods of identifying cells with modified genomes.
摘要翻译：提供了组合物和方法，用于产生和促进细胞内基因组的双向靶向修饰和用于产生包含经修饰的基因组的非人动物。还提供了用于修饰细胞内基因组的组合物和方法，所述基因组对于等位基因是杂合的，以使该等位基因变得纯合。这些方法利用Cas蛋白和两个或更多个靶向相同基因组目标基因座内不同位置的引导RNA。还提供了鉴定具有修饰基因组的细胞的方法。

7. 发明申请

WO2016061374A1 METHODS AND COMPOSITIONS FOR GENERATING OR MAINTAINING PLURIPOTENT CELLS 审中-公开
标题翻译：用于产生或维持PLUIPOTENT细胞的方法和组合物
公开(公告)号：WO2016061374A1
公开(公告)日：2016-04-21
申请号：PCT/US2015/055776
申请日：2015-10-15
申请人： REGENERON PHARMACEUTICALS, INC.
发明人： KUNO, Junko , AUERBACH, Wojtek , VALENZUELA, David M.
IPC分类号： C12N5/074 , C12N5/00
CPC分类号： C12N5/0696 , C12N2500/32 , C12N2500/38 , C12N2500/44 , C12N2500/60 , C12N2501/235 , C12N2501/727 , C12N2509/00
摘要： Methods and compositions are provided for generating or maintaining human iPS cells in culture. Methods include the use of a low osmolality medium to make human iPS cells, or use of a low osmolality medium to maintain human iPS cells. Methods for making targeted genetic modification to human iPS cells cultured in low osmolality medium are also included. Compositions include human iPS cells cultured and maintained using the low osmolality medium defined herein.
摘要翻译：提供了用于在培养物中产生或维持人iPS细胞的方法和组合物。方法包括使用低渗透浓度培养基制备人iPS细胞，或使用低渗透浓度培养基来维持人类iPS细胞。还包括对在低渗透浓度培养基中培养的人iPS细胞进行靶向遗传修饰的方法。组合物包括使用本文定义的低渗透浓度培养基培养和维持的人iPS细胞。

8. 发明申请

WO2015200805A3 METHODS AND COMPOSITIONS FOR TARGETED GENETIC MODIFICATIONS AND METHODS OF USE 审中-公开
公开(公告)号：WO2015200805A3
公开(公告)日：2015-12-30
申请号：PCT/US2015/038001
申请日：2015-06-26
申请人： REGENERON PHARMACEUTICALS, INC.
发明人： FRENDEWEY, David , DROGUETT, Gustavo , GAGLIARDI, Anthony , KUNO, Junko , AUERBACH, Wojtek , VALENZUELA, David M.
IPC分类号： C12N5/0735 , C12N15/873 , C12N15/85 , C12N15/90
摘要： Methods and compositions are provided for generating targeted genetic modifications on the Y chromosome or a challenging target locus. Compositions include an in vitro culture comprising an XY pluripotent and/or totipotent animal cell (i.e., XY ES cells or XY iPS cells) having a modification that decreases the level and/or activity of an Sry protein; and, culturing these cells in a medium that promotes development of XY F0 fertile females. Such compositions find use in various methods for making a fertile female XY non-human mammal in an F0 generation.

9. 发明申请

WO2015088643A1 METHODS AND COMPOSITIONS FOR THE TARGETED MODIFICATION OF A GENOME 审中-公开
标题翻译：基因组目标修饰的方法和组合
公开(公告)号：WO2015088643A1
公开(公告)日：2015-06-18
申请号：PCT/US2014/060788
申请日：2014-10-15
申请人： REGENERON PHARMACEUTICALS, INC.
发明人： FRENDEWEY, David , AUERBACH, Wojtek , LAI, Ka-Man Venus , MUJICA, Alexander O. , LEE, Jeffrey D. , DROGUETT, Gustavo , TRZASKA, Sean , HUNT, Charleen , GAGLIARDI, Anthony , KUNO, Junko , VALENZUELA, David M. , YANCOPOULOS, George D.
IPC分类号： C12N15/85 , C12N9/22
CPC分类号： C12N15/8509 , A01K67/0276 , A01K67/0278 , A01K2217/072 , A01K2217/075 , A01K2227/105 , A01K2267/0362 , A01K2267/0387 , C12N9/22 , C12N15/1024 , C12N15/85 , C12N15/907 , C12N2015/8527 , C12N2800/40 , C12N2810/00 , C12N2810/10 , C12N2810/40 , C12N2999/007
摘要： Compositions and methods are provided for modifying a genomic locus of interest in a eukaryotic cell, a mammalian cell, a human cell or a non-human mammalian cell using a large targeting vector (LTVEC) comprising various endogenous or exogenous nucleic acid sequences as described herein. Further methods combine the use of the LTVEC with a CRISPR/Cas system. Compositions and methods for generating a genetically modified non-human animal comprising one or more targeted genetic modifications in their germline are also provided.
摘要翻译：提供组合物和方法用于使用包含本文所述的各种内源或外源核酸序列的大靶向载体（LTVEC）修饰真核细胞，哺乳动物细胞，人细胞或非人哺乳动物细胞中的感兴趣的基因组座位。。其他方法将LTVEC与CRISPR / Cas系统的使用相结合。还提供了用于产生遗传修饰的非人动物的组合物和方法，其包含其种系中的一种或多种靶向遗传修饰。

10. 发明申请

WO2015021298A3 LINCRNA-DEFICIENT NON-HUMAN ANIMALS 审中-公开
公开(公告)号：WO2015021298A3
公开(公告)日：2015-02-12
申请号：PCT/US2014/050178
申请日：2014-08-07
申请人： REGENERON PHARMACEUTICALS, INC. , PRESIDENT AND FELLOWS OF HARVARD COLLEGE
发明人： LAI, Ka-man Venus , GONG, Guochun , RINN, John , FRENDEWEY, David , VALENZUELA, David M.
IPC分类号： C12N15/00 , C12N15/87 , A61K48/00
摘要： Genetically modified non-human animals are provided that exhibit a functional lack of one or more lncRNAs. Methods and compositions for disrupting, deleting, and/or replacing lncRNA-encoding sequences are provided. Genetically modified mice that age prematurely are provided. Also provided are cells, tissues and embryos that are genetically modified to comprise a loss-of-function of one or more lncRNAs.

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