NON-HUMAN ANIMALS COMPRISING A HUMANIZED COAGULATION FACTOR 12 LOCUS

Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized coagulation factor XII (F12) 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 com...

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Main Authors MACDONALD, Lynn, CHALOTHORN, Dan, MITNAUL, Lyndon, MORTON, Lori, ZAMOLODCHIKOV, Daria, TANG, Yajun, ALESSANDRI-HABER, Nicole
Format Patent
LanguageEnglish
Published 29.09.2021
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Summary:Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized coagulation factor XII (F12) 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 F12 locus express a human coagulation factor XII protein or a chimeric coagulation factor XII protein, fragments of which are from human coagulation factor XII. Methods are provided for using such non-human animals comprising a humanized F12 locus to assess in vivo efficacy of human-coagulation-factor-XII-targeting reagents such as nuclease agents designed to target human F12. A short isoform of F12 that is produced locally in the brain, and methods of using the short isoform, are also provide.
Bibliography:Application Number: SG11202108454R