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基因治疗的原理、工具与应用解析
慢病毒载体特别适用于治疗血液系统疾病和遗传性疾病,如β-地中海贫血和X-连锁重症联合免疫缺陷症(SCID)。 图6.慢病毒载体的主要临床用途 [5]。a.原发性免疫缺陷的矫正。使用病毒载体传递共同γ链(γc),可以恢复SCID-X1患者的免疫功能。b. 肿瘤特异性T细胞 ...
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