Restoration of lymphoid populations in a murine model of X-linked severe combined immunodeficiency by a gene-therapy approach.
Restoration of lymphoid populations in a murine model of X-linked severe combined immunodeficiency by a gene-therapy approach.
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通过基因治疗方法恢复 X 连锁严重联合免疫缺陷小鼠模型中的淋巴群体。
DOI：
10.1182/blood.v94.9.3027
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发表时间：
1999
期刊：
影响因子：
20.3
通讯作者：
Warren J. Leonard
中科院分区：
文献类型：
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作者：
Mindy S. Lo;Michael L. Bloom;Kazunori Imada;Maria Berg;Julie Bollenbacher;Eda T. Bloom;B. Kelsall;Warren J. Leonard
X-linked severe combined immunodeficiency (XSCID) is a life-threatening syndrome in which both cellular and humoral immunity are profoundly compromised. This disease results from mutations in the IL2RG gene, which encodes the common cytokine receptor gamma chain, gamma(c). Previously, we generated gamma(c)-deficient mice as a murine model of XSCID. We have now used lethally irradiated gamma(c)-deficient mice to evaluate a gene therapeutic approach for treatment of this disease. Transfer of the human gamma(c) gene to repopulating hematopoietic stem cells using an ecotropic retrovirus resulted in an increase in T cells, B cells, natural killer (NK) cells, and intestinal intraepithelial lymphocytes, as well as normalization of the CD4:CD8 T-cell ratio and of serum Ig levels. In addition, the restored cells could proliferate in response to interleukin-2 (IL-2). Thus, our results provide added support that gene therapy is a feasible therapeutic strategy for XSCID. Moreover, because we used a vector directing expression of human gamma(c) to correct a defect in gamma(c)-deficient mice, these data also indicate that human gamma(c) can cooperate with the distinctive cytokine receptor chains such as IL-2Rbeta and IL-7Ralpha to mediate responses to murine cytokines in vivo.
H.Otani：“白细胞介素 (IL) -2 和 IL-3 在用人 IL-2 受体 β 链转导的鼠 IL-3 依赖性 32D 细胞中诱导不同但重叠的反应：除 p56 之外的酪氨酸激酶的参与^
DOI：
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发表时间：
--
期刊：
影响因子：
--
作者：
通讯作者：
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影响因子：
30.8
作者：
Puel, A;Ziegler, SF;Leonard, WJ
通讯作者：
Leonard, WJ
SR α启动子：一种高效、多功能的哺乳动物cDNA表达系统，由猿猴病毒40早期启动子和人T细胞白血病病毒1型长末端重复序列的R-U5片段组成
影响因子：
5.3
作者：
Y. Takebe;M. Seiki;J. Fujisawa;P. Hoy;K. Yokota;K. Arai;Mitsuaki Yoshida;N. Arai
通讯作者：
N. Arai