Genetic or acquired defects in FOXP3⁺ regulatory T cells (Tregs) play a key role in many immune‐mediated diseases including immune dysregulation polyendocrinopathy, enteropathy, X‐linked (IPEX) syndrome. Previously, we demonstrated CD4⁺ T cells from healthy donors and IPEX patients can be converted into functional Treg‐like cells by lentiviral transfer of FOXP3 (CD4^LVFOXP3). These CD4^LVFOXP3 cells have potent regulatory function, suggesting their potential as an innovative therapeutic. Here, we present molecular and preclinical in vivo data supporting CD4^LVFOXP3 cell clinical progression.

中文翻译：

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人类工程改造的 Treg 样细胞抑制 FOXP3 缺陷型 T 细胞，但在体内保留适应性免疫反应

FOXP3 ⁺调节性 T 细胞 (Tregs) 的遗传或获得性缺陷在许多免疫介导疾病中发挥关键作用，包括免疫失调多内分泌病、肠病、X 连锁 (IPEX) 综合征。此前，我们证明了来自健康供体和 IPEX 患者的 CD4 ^{+ T 细胞可以通过}FOXP3（CD4 ^LVFOXP3）的慢病毒转移转化为功能性 Treg 样细胞。这些 CD4 ^LVFOXP3细胞具有强大的调节功能，表明它们具有作为创新疗法的潜力。在这里，我们提供了支持 CD4 ^LVFOXP3细胞临床进展的分子和临床前体内数据。