Engineered lipid nanoparticles for in vivo and durable editing of haematopoietic stem cells within humanized mice.

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DOI:10.1038/s41551-026-01765-w
作者：Jingjing Du,?Zijin Luo,?Dan Xie,?Yan Chen,?Ming Yang,?Qiang Li,?Lisha Wang,?Lu Han,?Yiming Zhang,?Haiwei Li,?Zilin Lan,?Haoping Shi,?Yinghui Li,?Qiang Cheng,?Fang Dong,?Yingdai Gao,?Yao Yao,?Tao Cheng
文献类型：期刊论文
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Nature

Abstract：In vivo genetic engineering of haematopoietic stem and progenitor cells (HSPCs) holds the potential to revolutionize the treatment landscape for numerous diseases. However, despite its trans

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