Precision BioSciences Presents Preclinical Data Highlighting PBGENE-PMM as a Potential Therapy for Primary Mitochondrial Myopathy

PBGENE-PMM项目介绍 - Precision BioSciences公司利用其ARCUS®平台开发PBGENE-PMM项目，旨在治疗m.3243相关的原发性线粒体肌病(PMM) [1] - ARCUS能够精确编辑线粒体DNA，消除突变线粒体DNA，保留正常功能的野生型线粒体DNA，从而改善线粒体功能 [2] - PBGENE-PMM在细胞中成功实现m.3243A>G杂合体内突变线粒体DNA的显著减少，进而提高基础和最大呼吸率 [3]