学术中心变态反应、哮喘病与免疫学

“ 气泡儿 童 ”是什么病?基因治疗为“气泡儿童”带来了希望!

“气泡儿童”发病的原因是他们的腺苷脱氨酶(ADA)基因缺陷导致嘌呤核苷代谢障碍,从而使T细胞和B细胞的分裂和成熟受到严重影响,表现为严重联合免疫缺陷(SCID),没有任何抵抗力,意味着一次小小的感冒都可能夺去孩子的生命,因此他们只能在气泡形的无菌、消毒的隔离舱中生存。骨髓移植是治疗“气泡儿童”的手段之一,可成功率过低。基因疗法作为一种替代手段而被寄予...

Published 2016-12-21
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