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NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS COMPRISING A V30M MUTATION AND METHODS OF USE
NON-HUMAN ANIMALS COMPRISING A HUMANIZED TTR LOCUS COMPRISING A V30M MUTATION AND METHODS OF USE
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机译:非人类动物,包含含有V30M突变和使用方法的人源化TTR基因座
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摘要
Non-human animal genomes, non-human animal cells, and non-human animals comprising a humanized TTR locus comprising a V30M mutation 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 TTR locus express a human TTR protein or a chimeric TTR protein, fragments of which are from human TTR. 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.
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