iPSCG6PD

Chaiwat Netsawang1, Malinee Tongbaen1, Natee Jearawiriyapaisarn2

  • 1Programme in Clinical Hematology Sciences, Department of Clinical Microscopy, Faculty of Allied Health Sciences, Chulalongkorn University, Bangkok, Thailand.

Scientific reports
|August 18, 2025
PubMed
概括

首次编辑有效地纠正了细胞系中普遍存在的G6PD Viangchan突变. 这种基因编辑工具显示了高精度和最小的副产品,为葡萄糖-6-酸盐脱酶缺乏症提供了一个有前途的治疗策略.