Senckenberg Institute of Pathology, Goethe-University Hospital Frankfurt, Frankfurt am Main, Germany.
Mol Med. 2011;17(11-12):1223-32. doi: 10.2119/molmed.2010.00193. Epub 2011 Jul 27.
Several cases of T-cell leukemia caused by gammaretroviral insertional mutagenesis in children treated for x-linked severe combined immunodeficiency (SCID) by transplantation of autologous gene-modified stem cells were reported. In a comparative analysis, we recently showed that mature T cells, on the contrary, are highly resistant to transformation by gammaretroviral gene transfer. In the present study, we observed immortalization of a single T-cell clone in vitro after gammaretroviral transduction of the T-cell protooncogene LMO2. This clone was CD4/CD8 double-negative, but expressed a single rearranged T-cell receptor. The clone was able to overgrow nonmanipulated competitor T-cell populations in vitro, but no tumor formation was observed after transplantation into Rag-1 deficient recipients. The retroviral integration site (RIS) was found to be near the IL2RA and IL15RA genes. As a consequence, both receptors were constitutively upregulated on the RNA and protein level and the immortalized cell clone was highly IL-2 dependent. Ectopic expression of both, the IL2RA chain and LMO2, induced long-term growth in cultured primary T cells. This study demonstrates that insertional mutagenesis can contribute to immortalization of mature T cells, although this is a rare event. Furthermore, the results show that signaling of the IL-2 receptor and the protooncogene LMO2 can act synergistically in maligniant transformation of mature T lymphocytes.
已报道几例因采用自体基因修饰干细胞移植治疗 X 连锁严重联合免疫缺陷(SCID)而导致 T 细胞白血病的病例,这些病例由γ逆转录病毒插入突变引起。在一项对比分析中,我们最近表明,成熟 T 细胞相反,对γ逆转录病毒基因转移的转化具有高度抗性。在本研究中,我们观察到在 T 细胞原癌基因 LMO2 的γ逆转录病毒转导后,体外单个 T 细胞克隆的永生化。该克隆 CD4/CD8 双阴性,但表达单个重排的 T 细胞受体。该克隆能够在体外过度生长非操作的竞争 T 细胞群体,但在移植入 Rag-1 缺陷受体后未观察到肿瘤形成。发现逆转录病毒整合位点(RIS)靠近 IL2RA 和 IL15RA 基因。因此,两种受体在 RNA 和蛋白质水平上均被持续上调,永生化细胞克隆对 IL-2 高度依赖。两种受体,即 IL2RA 链和 LMO2 的异位表达,诱导培养的原代 T 细胞的长期生长。这项研究表明,插入突变可导致成熟 T 细胞的永生化,尽管这是一种罕见事件。此外,结果表明 IL-2 受体和原癌基因 LMO2 的信号可以协同作用于成熟 T 淋巴细胞的恶性转化。