Regeneron Pharmaceuticals, Inc.

Jeffery Haines

Andrew J Murphy

David Frendewey

Brian Zambrowicz

Meghan Drummond Samuelson

Suzanne Hartford

Keith Crosby

Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TTR locus 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 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.

Non-human animals comprising a humanized TTR locus and methods of use

Non-human animal cells and non-human animals comprising a humanized TTR locus comprising a beta-slip mutation and methods of using such non-human animal cells and non-human animals are provided. Non-human animal cells or non-human animals comprising a humanized TTR locus comprising a beta-slip mutation 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 comprising a humanized TTR locus to assess in vivo efficacy of human-TTR-targeting reagents such as nuclease agents designed to target human TTR.

Mouse comprising a humanized TTR locus with a beta-slip mutation and methods of use

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New York, NY, United States of America

Jeffery Haines