Exon 2通过CRISPR/Cas9技术用两个sgRNAs(针对ATCTTGGCCCGATGTTTAGC和GGCTAAACATCGGGCCAAGA)进行了靶向,产生了10个碱基的缺失(GATGGACGAT),导致了移码和早停密码子。(来源:J:299228)
Legend:
cx: complex: > 1 genome feature ot: other: hemizygous, indeterminate,... (F): Female
(M): Male
N: normal phenotype
(#): related diseases count