T-LYMPHOCYTE-DIRECTED GENE-THERAPY FOR ADA(-) SCID - INITIAL TRIAL RESULTS AFTER 4 YEARS
T-LYMPHOCYTE-DIRECTED GENE-THERAPY FOR ADA(-) SCID - INITIAL TRIAL RESULTS AFTER 4 YEARS
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DOI：
10.1126/science.270.5235.475
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发表时间：
1995-10-20
期刊：
影响因子：
56.9
通讯作者：
ANDERSON, WF
中科院分区：
文献类型：
--
作者：
BLAESE, RM;CULVER, KW;ANDERSON, WF
1990 年，开始了一项临床试验，利用逆转录病毒介导将腺苷脱氨酶 (ADA) 基因转移到两名患有严重联合免疫缺陷 (ADA(-) SCID) 的儿童的 T 细胞中。血液 T 细胞的数量以及许多细胞和体液免疫反应都恢复正常。基因治疗在 2 年后结束，但 T 细胞中的整合载体和 ADA 基因表达仍然存在。尽管许多组成部分仍有待完善，但本文得出的结论是，对于某些患有这种严重免疫缺陷疾病的患者来说，基因疗法可以是一种安全有效的治疗补充。
In 1990, a clinical trial was started using retroviral-mediated transfer of the adenosine deaminase (ADA) gene into the T cells of two children with severe combined immunodeficiency (ADA(-) SCID). The number of blood T cells normalized as did many cellular and humoral immune responses. Gene treatment ended after 2 years, but integrated vector and ADA gene expression in T cells persisted. Although many components remain to be perfected, it is concluded here that gene therapy can be a safe and effective addition to treatment for some patients with this severe immunodeficiency disease.