Base Editing of HBG1 and HBG2 Promoters for Sickle Cell Disease.

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DOI:10.1056/NEJMoa2504835
文献类型：期刊论文
补充材料：只需要正文
文献链接：https://doi.org/10.1056/nejmoa2504835

New England Journal of Medicine (NEJM)

Abstract：BACKGROUND Sickle cell disease is characterized by chronic hemolytic anemia and recurrent severe vaso-occlusive crises. Ristoglogene autogetemcel (risto-cel) includes autologous CD34+ hemat

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