Clinically translatable mitochondrial gene therapy in muscle using tandem zinc finger nuclease architecture

We studied the use of tandem zinc finger nucelase arrangements delivered by AAV in a mouse model of mitochondrial genetic disease. Overall design: Assessments of nuclear DNA off-targets was analysed by Illumina NGS

本研究探讨了腺相关病毒（Adeno-Associated Virus, AAV）递送的串联锌指核酸酶组合在线粒体遗传疾病小鼠模型中的应用。总体实验设计：通过Illumina NGS对核DNA脱靶位点的评估结果进行分析。