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造血干细胞（Hematopoietic stem cell，HSC）基因治疗是治疗造血系统遗传疾病的创新疗法，如镰状细胞病、严重联合免疫缺陷和范可尼贫血等。该疗法通过修饰或引入核苷酸序列到 HSC 基因组来治愈疾病，现有多种技术可实现 HSC 基因组的遗传修饰，包括慢病毒载体 ...

A comprehensive understanding of in vivo heterogeneity and specificity of HSC responses to external stimuli is currently lacking. We performed single-cell RNA sequencing (scRNA-Seq) on functionally ...

A hallmark of adult hematopoiesis is the continuous replacement of blood cells with limited lifespans. While active hematopoietic stem cell (HSC) contribution to multilineage hematopoiesis is the ...