CRISPR/cas9基因编辑用来去除exon 2。选择的导向RNA针对exon 2上游的[AAGTGTCCCCAAATATTGTC; ATCCTTTCCTGACAATATTT]和下游的[GCACCTAGCACATTGCCATG; CACCTAGCACATTGCCATGT]序列。(来源:J:101977)
Legend:
cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count