Regeneron Pharmaceuticals, Inc.
NON-HUMAN ANIMALS COMPRISING A HUMANIZED PNPLA3 LOCUS AND METHODS OF USE

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-PNPLA3-targeting reagents such as nuclease agents designed to target human PNPLA3.