Abstract
CRISPR-Ca9 system is a newly developed gene-editing technology, which is widely used in biology and medical research. In this project, we want to knock-in a mutation found in a human pedigree into mice through CRISPR-Cas9 technology to validate its pathogenic effect. We download corresponding mice genomic sequences and design guide RNA and donor oligo sequences according to CRISPR-Cas9 target principles. Following experiments confirm that this set of sequences is effective in mice cell line.