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    NON-HUMAN ANIMALS HAVING A HEXANUCLEOTIDE REPEAT EXPANSION IN A C9ORF72 LOCUS
    2.
    发明申请
    NON-HUMAN ANIMALS HAVING A HEXANUCLEOTIDE REPEAT EXPANSION IN A C9ORF72 LOCUS 审中-公开
    在C9ORF72基因座中具有双核苷酸重复扩增的非人动物

    公开(公告)号：WO2018064600A1

    公开(公告)日：2018-04-05

    申请号：PCT/US2017/054551

    申请日：2017-09-29

    Applicant: REGENERON PHARMACEUTICALS, INC.

    Inventor： HESLIN, David ,  ALLY, Roxanne ,  SIAO, Chia-Jen ,  LAI, Ka-Man, Venus ,  VALENZUELA, David, M. ,  GUO, Chunguang ,  LACROIX-FRALISH, Michael ,  MACDONALD, Lynn ,  SHARMA, Aarti ,  KAJIMURA, Daisuke ,  DROGUETT, Gustavo ,  FRENDEWEY, David

    IPC: A01K67/027 ,  C12N15/90 ,  C12N15/113

    Abstract: A non-human animal (e.g., a rodent) model for diseases associated with a C9ORF72 heterologous hexanucleotide repeat expansion sequence is provided, which non-human animal comprises a heterologous hexanucleotide repeat (GGGGCC) in an endogenous C9ORF72 locus. A non-human animal disclosed herein comprising a heterologous hexanucleotide repeat expansion sequence comprising at least one instance, e.g., repeat, of a hexanucleotide (GGGGCC) sequence may further exhibit a characteristic and/or phenotype associated with one or more neurodegenerative disorders (e.g., amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD), etc.). 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)) are also provided.

    Abstract translation: 提供了与C9ORF72异源六核苷酸重复扩增序列相关的疾病的非人动物（例如啮齿动物）模型，所述非人动物包含异源六聚核苷酸 在内源性C9ORF72基因座中重复（GGGGCC）。 本文公开的包含包含六核苷酸（GGGGCC）序列的至少一个实例，例如重复序列的异源六核苷酸重复扩增序列的非人动物还可以表现出与一种或多种神经变性病症（例如， 肌萎缩侧索硬化（ALS）和/或额颞叶痴呆（FTD）等）。 还提供了可用于预防，延缓或治疗一种或多种神经变性（例如，肌萎缩侧索硬化（ALS，也称为Lou Gehrig病）和额颞痴呆（FTD））的治疗候选物的方法。

    SELECTION OF PLURIPOTENT CELLS FOR PRODUCTION OF FERTILE XY FEMALE MICE
    6.
    发明申请
    SELECTION OF PLURIPOTENT CELLS FOR PRODUCTION OF FERTILE XY FEMALE MICE 审中-公开
    选择用于生产可食用X射线母鼠的小鼠细胞

    公开(公告)号：WO2017049240A1

    公开(公告)日：2017-03-23

    申请号：PCT/US2016/052345

    申请日：2016-09-16

    Applicant: REGENERON PHARMACEUTICALS, INC.

    Inventor： SCHMAHL, Jennifer ,  FRENDEWEY, David ,  KUNO, Junko ,  SIAO, Chia-Jen ,  DROGUETT, Gustavo ,  BAI, Yu ,  AUERBACH, Wojtek

    IPC: A01K67/027 ,  C12N5/0735

    CPC classification number: A01K67/0271 ,  A01K2207/12 ,  A01K2207/35 ,  A01K2227/105 ,  A01K2267/02 ,  C12N5/0606 ,  C12Q1/6876 ,  C12Q2600/136 ,  C12Q2600/156 ,  C12Q2600/158 ,  G01N33/5073

    Abstract: Methods and compositions are provided for generating F0 fertile XY female animals. The methods and compositions involve making XY pluripotent or totipotent animal cells, in vitro cell cultures, or embryos that are capable of producing a fertile female XY animal in an F0 generation. Such cells, embryos, and animals can be made by silencing a region of the Y chromosome. Optionally, the cells can also be cultured in feminizing medium such as a low-osmolality medium and/or can be modified to decrease the level and/or activity of an Sry protein. Methods and compositions are also provided for silencing a region of the Y chromosome in an XY pluripotent or totipotent animal cell, or in vitro cell cultures, embryos, or animals derived therefrom, by maintaining an XY pluripotent or totipotent animal cell in a feminizing medium. Methods and compositions are also provided for maintaining a population of XY pluripotent or totipotent animal cells in a feminizing medium and selecting cells or clones having increased capabilities for producing a fertile female XY animal in an F0 generation. Methods and compositions are also provided for screening for compounds with feminizing activity or for optimizing concentrations of components in feminizing media.

    Abstract translation: 提供了用于产生F0可育XY雌性动物的方法和组合物。 所述方法和组合物包括制备XY多能或全能动物细胞，体外细胞培养物或能够在F0代中产生可育雌性XY动物的胚胎。 可以通过沉默Y染色体的区域来制备这样的细胞，胚胎和动物。 任选地，细胞还可以在女性化培养基如低渗透浓度培养基中培养和/或可被修饰以降低Sry蛋白的水平和/或活性。 还提供了通过将XY多能或全能动物细胞维持在女性化培养基中，从而在XY多能或全能动物细胞或体外细胞培养物，胚胎或由其衍生的动物中沉默Y染色体区域的方法和组合物。 还提供了用于在女性化培养基中维持XY多能或全能动物细胞群的方法和组合物，并选择具有增加在F0代中产生可育雌性XY动物的能力的细胞或克隆。 还提供了用于筛选具有女性化活性的化合物或优化女性化培养基中组分浓度的方法和组合物。

    SELECTION OF PLURIPOTENT CELLS FOR PRODUCTION OF FERTILE XY FEMALE MICE
    8.
    发明申请
    SELECTION OF PLURIPOTENT CELLS FOR PRODUCTION OF FERTILE XY FEMALE MICE 审中-公开

    公开(公告)号：WO2017049240A8

    公开(公告)日：2017-03-23

    申请号：PCT/US2016/052345

    申请日：2016-09-16

    Applicant: REGENERON PHARMACEUTICALS, INC.

    Inventor： SCHMAHL, Jennifer ,  FRENDEWEY, David ,  KUNO, Junko ,  SIAO, Chia-Jen ,  DROGUETT, Gustavo ,  BAI, Yu ,  AUERBACH, Wojtek

    IPC: A01K67/027 ,  C12N5/0735

    Abstract: Methods and compositions are provided for generating F0 fertile XY female animals. The methods and compositions involve making XY pluripotent or totipotent animal cells, in vitro cell cultures, or embryos that are capable of producing a fertile female XY animal in an F0 generation. Such cells, embryos, and animals can be made by silencing a region of the Y chromosome. Optionally, the cells can also be cultured in feminizing medium such as a low-osmolality medium and/or can be modified to decrease the level and/or activity of an Sry protein. Methods and compositions are also provided for silencing a region of the Y chromosome in an XY pluripotent or totipotent animal cell, or in vitro cell cultures, embryos, or animals derived therefrom, by maintaining an XY pluripotent or totipotent animal cell in a feminizing medium. Methods and compositions are also provided for maintaining a population of XY pluripotent or totipotent animal cells in a feminizing medium and selecting cells or clones having increased capabilities for producing a fertile female XY animal in an F0 generation. Methods and compositions are also provided for screening for compounds with feminizing activity or for optimizing concentrations of components in feminizing media.

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