使用sgRNAs和ssODN模板,通过CRISPR/Cas9技术,该基因被修改,使其同源区段类似于人类的C4A基因:内源性密码子序列PCPVIH被替换为PCPVLD(突变位点为p.I1120_H1121delLDins,参考文献:J:301308)。
Legend:
cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count