NON-HUMAN ANIMALS MODELS OF RETINOSCHISIS

    公开(公告)号:US20210307304A1

    公开(公告)日:2021-10-07

    申请号:US17348907

    申请日:2021-06-16

    Abstract: Non-human animals suitable for use as animal models for Retinoschisis are provided. In some embodiments, provided non-human animals are characterized by a disruption in a Retinoschisin-1 locus. In some embodiments, provided non-human animals are characterized by a mutant Retinoschisin-1 gene. The non-human animals may be described, in some embodiments, as having a phenotype that includes the development of one or more symptoms or phenotypes associated with Retinoschisis. Methods of identifying therapeutic candidates that may be used to prevent, delay or treat Retinoschisis or eye-related diseases, disorders or conditions are also provided.

    NON-HUMAN ANIMALS COMPRISING A HUMANIZED KLKB1 LOCUS AND METHODS OF USE

    公开(公告)号:US20230081547A1

    公开(公告)日:2023-03-16

    申请号:US17759545

    申请日:2021-02-05

    Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized KLKB1 locus and methods of making and 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 KLKB1 locus express a human plasma kallikrein protein or a chimeric plasma kallikrein protein, fragments of which are from human plasma kallikrein. Methods are provided for using such non-human animals comprising a humanized KLKB1 locus to assess in vivo efficacy of human-KLKB 1-targeting reagents such as nuclease agents designed to target human KLKB1.

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