CRISPR/Cas9技术在exon 3的两侧引入了loxP位点,并将exon 4的5'端移动了两个碱基到exon 3的3'端,以便在条件性删除exon 3时产生框架移位。测序结果显示,工程化的exon-3和exon-4连接处正常剪接,生成的mRNA与野生型小鼠的mRNA完全一致。在纯合子和野生型小鼠间,mRNA水平相当。(来源:J:271070)
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cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count