NGS on the 5BP correction followed by genome editing therapy

The studied mouse model have a 5BP deletion in the RPGR gene and CRISPR/Cas9-mediated in vivo genome editing therapy was designed to repair it. The next-generation sequencing is done to verify the 5BP correction.The results show homology-directed repair in the target site.

本研究所使用的小鼠模型在RPGR基因中存在5个碱基对（5BP）的缺失。本研究设计了CRISPR/Cas9介导的体内基因组编辑疗法以修复该基因缺失。研究采用下一代测序（next-generation sequencing）技术验证该5BP位点的修复效果。结果显示靶位点发生了同源定向修复（homology-directed repair）。