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In vivo genome editing of human haematopoietic stem cells for treatment of blood disorders using mRNA delivery

Saijuan Xu, Dan Liang, Qiudao Wang, Yan Cheng, Da Xie, Gui Yang, Haokun Zhang, Changrui Feng, Feiyan Zhao, Wendan Ren, Gongrui Sun, Yang Yang, Lin Li, Yongrong Lai, Bin Fu, Yuming Lu, Zi Jun Wang, Yuxuan Wu

2025Nature Biomedical Engineering9 citationsDOI

Topics & Concepts

HaematopoiesisStem cellGenome editingGenetic enhancementIn vivoBiologyGene deliveryEx vivoCell biologyImmunologyGeneGenomeGeneticsRNA Interference and Gene DeliveryCRISPR and Genetic EngineeringVirus-based gene therapy research
In vivo genome editing of human haematopoietic stem cells for treatment of blood disorders using mRNA delivery | Litcius