NON-HUMAN ANIMALS COMPRISING A HUMANIZED PNPLA3 LOCUS AND METHODS OF USE

    公开(公告)号:WO2021154791A1

    公开(公告)日:2021-08-05

    申请号:PCT/US2021/015192

    申请日:2021-01-27

    Abstract: Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized PNPLA3 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 PNPLA3 locus express a human PNPLA3 protein or a chimeric PNPLA3 protein, fragments of which are from human PNPLA3. Methods are provided for using such non-human animals comprising a humanized PNPLA3 locus to assess in vivo efficacy of human -PNPL A3 -targeting reagents such as nuclease agents designed to target human PNPLA3.

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