The importance of microbial communities to human and environmental health has motivated researchers to develop methods for the efficient characterization of these communities. The most common and cost ...

本研究针对腺苷脱氨酶2缺乏症(DADA2)这一罕见遗传病，探索了慢病毒载体(LV)介导的ADA2基因疗法的临床前效果。研究人员通过人源化NBSGW小鼠模型，证实LV-ADA2转导的健康供体和患者来源造血干细胞(HSC)能保持多系分化能力，且未在非造血器官检测到病毒整合。