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    CRISPR/CAS-MEDIATED UPREGULATION OF HUMANIZED TTR ALLELE
    1.
    发明申请
    CRISPR/CAS-MEDIATED UPREGULATION OF HUMANIZED TTR ALLELE 审中-公开

    公开(公告)号：WO2021108363A1

    公开(公告)日：2021-06-03

    申请号：PCT/US2020/061943

    申请日：2020-11-24

    Applicant: REGENERON PHARMACEUTICALS, INC.

    Inventor： HUNT, Charleen ,  HARTFORD, Suzanne ,  GONG, Guochun ,  HAINES, Jeffery ,  ZAMBROWICZ, Brian

    IPC: A01K67/027 ,  C12N15/11 ,  C07K14/435

    Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TTR locus and CRISPR/Cas synergistic activation mediator system components and methods of using such non-human animal genomes, non-human animal cells, and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TTR locus express a human transthyretin protein or a chimeric transthyretin protein, fragments of which are from human transthyretin. Methods are provided for using such non-human animals to increase expression of the humanized TTR locus in vivo. Methods are provided for using such non-human animals comprising a humanized TTR locus to assess in vivo efficacy of human-TTR-targeting reagents such as nuclease agents designed to target human TTR. Methods are also provided for making such non-human animals comprising a humanized TTR locus and CRISPR/Cas synergistic activation mediator system components.

    CRISPR REPORTER NON-HUMAN ANIMALS AND USES THEREOF
    2.
    发明申请
    CRISPR REPORTER NON-HUMAN ANIMALS AND USES THEREOF 审中-公开

    公开(公告)号：WO2019028023A3

    公开(公告)日：2019-02-07

    申请号：PCT/US2018/044606

    申请日：2018-07-31

    Applicant: REGENERON PHARMACEUTICALS, INC.

    Inventor： GONG, Guochun ,  HUNT, Charleen ,  BRYDGES, Susannah ,  HARTFORD, Suzanne ,  FRENDEWEY, David ,  ZAMBROWICZ, Brian ,  MURPHY, Andrew J.

    IPC: A01K67/027

    Abstract: Methods and compositions are provided for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo or ex vivo. The methods and compositions employ non-human animals comprising a CRISPR reporter such as a genomically integrated CRISPR reporter for detecting and measuring targeted excision of a sequence between two CRISPR/Cas nuclease cleavage sites or disruption of a sequence near a CRISPR/Cas nuclease cleavage site and/or measuring CRISPR/Cas-induced recombination of the CRISPR reporter with an exogenous donor nucleic acid to convert the coding sequence for a first reporter protein to the coding sequence for a different second reporter protein. Methods and compositions are also provided for making and using these non-human animals.

    MICE COMPRISING MUTATIONS RESULTING IN EXPRESSION OF C-TRUNCATED FIBRILLIN-1
    3.
    发明申请
    MICE COMPRISING MUTATIONS RESULTING IN EXPRESSION OF C-TRUNCATED FIBRILLIN-1 审中-公开
    包含引起C-受体结合的FIBRILLIN-1表达的突变的小鼠

    公开(公告)号：WO2018023014A1

    公开(公告)日：2018-02-01

    申请号：PCT/US2017/044409

    申请日：2017-07-28

    Applicant: REGENERON PHARMACEUTICALS, INC.

    Inventor： HUNT, Charleen ,  MASTAITIS, Jason ,  GONG, Guochun ,  LAI, Ka-Man Venus ,  GROMADA, Jesper ,  ECONOMIDES, Aris, N.

    IPC: A01K67/027

    Abstract: Provided are non-human animals comprising a mutation in the Fbnl gene to model neonatal progeroid syndrome with congenital lipodystrophy (NPSCL). Also provided are methods of making such non-human animal models. The non-human animal models can be used for screening compounds for activity in inhibiting or reducing NPSCL or ameliorating NPSCL- like symptoms or screening compounds for activity potentially harmful in promoting or exacerbating NPSCL as well as to provide insights in to the mechanism of NPSCL and potentially new therapeutic and diagnostic targets.

    Abstract translation: 提供包含Fbnl基因中的突变的非人动物以模拟具有先天性脂肪代谢障碍（NPSCL）的新生儿原病综合征。 还提供了制造这种非人动物模型的方法。 非人类动物模型可用于筛选化合物抑制或减少NPSCL或改善NPSCL样症状的活性或筛选化合物的活性，以促进或加剧NPSCL的潜在危害，并提供对NPSCL机制的见解和 潜在的新的治疗和诊断目标。

    CAS-TRANSGENIC MOUSE EMBRYONIC STEM CELLS AND MICE AND USES THEREOF
    5.
    发明申请
    CAS-TRANSGENIC MOUSE EMBRYONIC STEM CELLS AND MICE AND USES THEREOF 审中-公开

    公开(公告)号：WO2019028032A1

    公开(公告)日：2019-02-07

    申请号：PCT/US2018/044615

    申请日：2018-07-31

    Applicant: REGENERON PHARMACEUTICALS, INC.

    Inventor： GONG, Guochun ,  HUNT, Charleen ,  KAJIMURA, Daisuke ,  HARTFORD, Suzanne ,  ZAMBROWICZ, Brian

    IPC: A01K67/027 ,  C12N15/90

    Abstract: Methods and compositions are provided herein for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo and ex vivo. The methods and compositions employ cells and non-human animals comprising a Cas expression cassette such as a genomically integrated Cas expression cassette so that the Cas protein can be constitutively available or available in a tissue- specific or temporal- specific manner. Methods and compositions are also provided for making and using these non-human animals, including use of these non-human animals to assess CRISPR/Cas activity in vivo via adeno-associated virus (AAV)-mediated delivery of guide RNAs to the non-human animals.

    CRISPR REPORTER NON-HUMAN ANIMALS AND USES THEREOF
    6.
    发明申请
    CRISPR REPORTER NON-HUMAN ANIMALS AND USES THEREOF 审中-公开

    公开(公告)号：WO2019028023A4

    公开(公告)日：2019-02-07

    申请号：PCT/US2018/044606

    申请日：2018-07-31

    Applicant: REGENERON PHARMACEUTICALS, INC.

    Inventor： GONG, Guochun ,  HUNT, Charleen ,  BRYDGES, Susannah ,  HARTFORD, Suzanne ,  FRENDEWEY, David ,  ZAMBROWICZ, Brian ,  MURPHY, Andrew J.

    IPC: A01K67/027

    Abstract: Methods and compositions are provided for assessing CRISPR/Cas-mediated non-homologous end joining (NHEJ) activity and/or CRISPR/Cas-induced recombination of a target genomic locus with an exogenous donor nucleic acid in vivo or ex vivo. The methods and compositions employ non-human animals comprising a CRISPR reporter such as a genomically integrated CRISPR reporter for detecting and measuring targeted excision of a sequence between two CRISPR/Cas nuclease cleavage sites or disruption of a sequence near a CRISPR/Cas nuclease cleavage site and/or measuring CRISPR/Cas-induced recombination of the CRISPR reporter with an exogenous donor nucleic acid to convert the coding sequence for a first reporter protein to the coding sequence for a different second reporter protein. Methods and compositions are also provided for making and using these non-human animals.

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