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23 天
疾病重点: X 连锁严重联合免疫缺陷 (X-SCID)
由 x 染色体上的 IL2RG 基因突变引起的严重联合免疫缺陷(SCID-X1 或“泡泡男孩病”)是一种毁灭性的遗传性疾病,导致男孩无法形成免疫系统。 如果他们暴露在环境中,即使是很短的时间,他们也会受到感染,[…] 一种单克隆抗体,可消耗血液干细胞并实现无 ...
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