A group around Kenichi NAGATA Generation has used an knock-in mouse model carrying a humanized Aβ sequence and edited the genome of the model zygotes using multiple combinations of CRISPR/Cas9 tools produces genetically mosaic animals with various App 3′-UTR deletions. An App knock-in mouse with a 34-bp deletion in a 52-bp regulatory element adjacent to the stop codon showed a substantial reduction in Aβ pathology, suggesting gene therapy as a putative future procedure to suppress amyloid ß production.

RIKEN news release, May 4, 2018