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健康捐献者的造血干细胞移植，可以治愈或改善各种非恶性疾病的病理，包括原发性免疫缺陷疾病、血红蛋白病、骨髓衰竭综合征和先天性代谢紊乱等。然而，未经化疗的患者干细胞移植失败的发生率较高，限制了其广泛应用。近日，匹兹堡大学的研究人员用脐带血成功治疗了儿童罕见遗传病，而且失败率大大降低。这项研究由匹兹堡大学医学中心匹兹堡儿童医院的Paul Szabolcs博士领导，并发表在《Blood Advances》上。
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