针对腺苷脱氨酶严重联合免疫缺陷病（ADA-SCID）的T淋巴细胞导向基因治疗：4年后的初步试验结果
T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years.
作者信息
Blaese R M, Culver K W, Miller A D, Carter C S, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P, Greenblatt J J, Rosenberg S A, Klein H, Berger M, Mullen C A, Ramsey W J, Muul L, Morgan R A, Anderson W F
机构信息
National Center for Human Genome Research, National Institutes of Health (NIH), Bethesda, MD 20892, USA.
出版信息
Science. 1995 Oct 20;270(5235):475-80. doi: 10.1126/science.270.5235.475.
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.
1990年，一项临床试验启动，将腺苷脱氨酶（ADA）基因通过逆转录病毒介导转移到两名重症联合免疫缺陷（ADA - SCID）儿童的T细胞中。血液中T细胞的数量恢复正常，许多细胞免疫和体液免疫反应也恢复正常。基因治疗在2年后结束，但T细胞中整合的载体和ADA基因表达持续存在。尽管许多方面仍有待完善，但本文得出结论，对于一些患有这种严重免疫缺陷疾病的患者，基因治疗可以成为一种安全有效的治疗补充手段。
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