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一个针对RUNX1的T细胞受体在急性髓性白血病中的框架转移突变

Nadine E Struckman¹, Georgia Koutsoumpli¹, Rob C M de Jong¹

¹Department of Hematology, Leiden University Medical Center, Leiden, The Netherlands.

|December 13, 2025

概括

针对瘤原RUNX1 (Runt相关转录因子1) 的替代阅读框架的免疫疗法在急性髓性白血病 (AML) 中显示出前景. 设计用于识别RUNX1新抗原的T细胞受体有效地杀死AML细胞,包括来自患者的样本和白血病干细胞.

科学领域:

在瘤学瘤学.
免疫学免疫学免疫学
分子生物学分子生物学

背景情况:

与Runt相关的转录因子1 (RUNX1) 对于造血细胞的分化至关重要.
RUNX1突变,特别是移突变,与急性髓性白血病 (AML) 的预后不佳有关.
这些框架转移突变可以导致瘤原蛋白转化为另一个读取框架.

研究的目的:

通过免疫疗法调查向瘤原RUNX1的替代阅读框架的潜力.
为了治疗向,识别和验证由RUNX1框架转移突变产生的新抗原.

主要方法:

引入了RUNX1框架转移突变到B细胞系中,并使用免疫组学识别了新.
查健康人群RUNX1新抗原特异性CD8T细胞使用-MHC四分体.
隔离和测序的T细胞受体 (TCR),然后为功能测定和体内研究设计CD8T细胞.

主要成果:

从RUNX1框架转移突变中鉴定出13种新.
孤立的T细胞克隆识别了4个HLA等位基因中的5个新抗原.
证明TCR工程T细胞有效地杀死RUNX1突变的AML细胞系,患者衍生的AML细胞和白血病干细胞在试验室和免疫缺陷小鼠中.

结论:

免疫疗法可以有效地向RUNX1框架转移突变.
基于TCR的免疫疗法具有治疗RUNX1-突变AML.患者的显著潜力.
这项研究验证了在AML治疗中针对替代阅读框架新抗原的概念.

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