Human I1061T NPC1 mice
TECHNOLOGY NUMBER: 2022-414
Technology No. 2022-414
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INNOVATION
CRISPR/Cas9 gene editing was used to insert human I1061T NPC1 cDNA into exon 2 of the mouse gene, immediately downstream of the mouse signal sequence. Gene targeted mice express human I1061T NPC1, but not mouse NPC1, and are a model of Niemann-Pick type C disease.