Generation of C9orf72h370 mice, an intron 1 humanised C9orf72 repeat-expansion knock-in model
R. Nair, Mireia Carcolé, David Thompson, Charlotte Tibbit, Ross McLeod, Alexander J. Cammack, Tatiana Jakubcova, Daniel Biggs, Matthew Wyles, Matthew T Parker, Adam Caulder, Lydia Teboul, Chloe L Fisher-Ward, Ali R Awan, Michael D. Flower, , Thomas J. Cunningham
The paper generates a mouse model with a humanised C9orf72 repeat expansion to study ALS and FTD, and demonstrates a method for creating such repeat expansions in genomic sequences.
bioRxiv · Feb 6, 2025 · PreprintMolecular Biology