CRISPR/Cas9通过同源定向修复(HDR)技术,在终止密码子前插入mCherry报告基因。使用两种sgRNAs,sgRNA1为ATTCTTATAGAGGACGCACT,sgRNA2为AAGTGCGTCCTCTATAAGAA,或者sgRNA1和sgRNA3的组合(AACATTGTGGTGGCTGATAT),目标定位在第4外显子和3'非编码区的连接处。(来源:J:302430)
Legend:
cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count