School of Pharmaceutical Sciences, University of São Paulo, Department of Clinical Analysis, Ribeirão Preto, São Paulo, Brazil.
Clin Exp Immunol. 2012 Jun;168(3):291-302. doi: 10.1111/j.1365-2249.2012.04583.x.
Type 1 diabetes (T1D) is a chronic autoimmune disease characterized by T cell-mediated destruction of pancreatic β cells, resulting in insulin deficiency and hyperglycaemia. Recent studies have described that apoptosis impairment during central and peripheral tolerance is involved in T1D pathogenesis. In this study, the apoptosis-related gene expression in T1D patients was evaluated before and after treatment with high-dose immunosuppression followed by autologous haematopoietic stem cell transplantation (HDI-AHSCT). We also correlated gene expression results with clinical response to HDI-AHSCT. We observed a decreased expression of bad, bax and fasL pro-apoptotic genes and an increased expression of a1, bcl-x(L) and cIAP-2 anti-apoptotic genes in patients' peripheral blood mononuclear cells (PBMCs) compared to controls. After HDI-AHSCT, we found an up-regulation of fas and fasL and a down-regulation of anti-apoptotic bcl-x(L) genes expression in post-HDI-AHSCT periods compared to pre-transplantation. Additionally, the levels of bad, bax, bok, fasL, bcl-x(L) and cIAP-1 genes expression were found similar to controls 2 years after HDI-AHSCT. Furthermore, over-expression of pro-apoptotic noxa at 540 days post-HDI-AHSCT correlated positively with insulin-free patients and conversely with glutamic acid decarboxylase autoantibodies (GAD65) autoantibody levels. Taken together, the results suggest that apoptosis-related genes deregulation in patients' PBMCs might be involved in breakdown of immune tolerance and consequently contribute to T1D pathogenesis. Furthermore, HDI-AHSCT modulated the expression of some apoptotic genes towards the levels similar to controls. Possibly, the expression of these apoptotic molecules could be applied as biomarkers of clinical remission of T1D patients treated with HDI-AHSCT therapy.
1 型糖尿病(T1D)是一种慢性自身免疫性疾病,其特征是 T 细胞介导的胰岛 β 细胞破坏,导致胰岛素缺乏和高血糖。最近的研究描述了中枢和外周耐受过程中细胞凋亡的损伤与 T1D 的发病机制有关。在这项研究中,我们评估了接受高剂量免疫抑制治疗后,再进行自体造血干细胞移植(HDI-AHSCT)治疗的 T1D 患者治疗前后的细胞凋亡相关基因表达,并将基因表达结果与 HDI-AHSCT 的临床反应相关联。我们观察到与对照组相比,患者外周血单核细胞(PBMCs)中促凋亡基因 bad、bax 和 fasL 的表达降低,而抗凋亡基因 a1、bcl-x(L) 和 cIAP-2 的表达增加。在 HDI-AHSCT 后,我们发现与移植前相比,post-HDI-AHSCT 时期 fas 和 fasL 的表达上调,而抗凋亡 bcl-x(L) 基因的表达下调。此外,HDI-AHSCT 后 2 年,bad、bax、bok、fasL、bcl-x(L) 和 cIAP-1 基因的表达水平与对照组相似。此外,HDI-AHSCT 后 540 天 noxa 的过表达与无胰岛素患者呈正相关,与谷氨酸脱羧酶自身抗体(GAD65)自身抗体水平呈负相关。总之,这些结果表明,患者 PBMCs 中细胞凋亡相关基因的失调可能与免疫耐受的破坏有关,并因此有助于 T1D 的发病机制。此外,HDI-AHSCT 调节了一些凋亡基因的表达,使其向与对照组相似的水平。可能,这些凋亡分子的表达可以作为接受 HDI-AHSCT 治疗的 T1D 患者临床缓解的生物标志物。