Micheli R, Pirovano S, Calandra G, Valotti M, Plebani A, Albertini A, Imberti L
Pediatric Neuropsychiatry, Spedali Civili of Brescia, Brescia, Italy.
Neuropediatrics. 2003 Jun;34(3):165-7. doi: 10.1055/s-2003-41280.
Ataxia-telangiectasia, a genetic disease caused by the homozygous mutation of the ATM gene, is frequently associated to a deficit of humoral and cellular immune functions. A decreased thymic output and skewed T cell and B cell receptor repertoires have been recently described in children over 7 years of age and in adults with this disease and have been proposed as a possible explanation for the immunodeficiency. To understand whether T cell defects arise early in life as a consequence of ATM gene mutations, we analysed the extent of thymic function by measuring the number of naïve T cells and by studying the heterogeneity of T cells by means of heteroduplex analysis, in two children less than 2 years old with a remarkable reduction of T cell count. We found that the thymic output is decreased in babies with ataxia-telangiectasia if compared with that observed in age-matched normal babies. The low production of new T cells is associated to a reduction of the diversity of alpha/beta, but not gamma/delta, T lymphocytes. Our data indicate that ATM mutation limits the generation of a wide alpha/beta T cell repertoire and this feature can be responsible for the immunodeficiency observed in ataxia-telangiectasia babies.
共济失调毛细血管扩张症是一种由ATM基因纯合突变引起的遗传性疾病,常伴有体液免疫和细胞免疫功能缺陷。最近在7岁以上儿童和患有这种疾病的成人中发现胸腺输出减少以及T细胞和B细胞受体库失衡,并被认为可能是免疫缺陷的一种解释。为了了解T细胞缺陷是否由于ATM基因突变在生命早期就已出现,我们通过测量幼稚T细胞数量来分析胸腺功能的程度,并通过异源双链分析研究T细胞的异质性,对象是两名2岁以下T细胞计数显著减少的儿童。我们发现,与年龄匹配的正常婴儿相比,患有共济失调毛细血管扩张症的婴儿胸腺输出减少。新T细胞的低产量与α/β(而非γ/δ)T淋巴细胞多样性的降低有关。我们的数据表明,ATM突变限制了广泛的α/βT细胞库的产生,这一特征可能是共济失调毛细血管扩张症婴儿中观察到的免疫缺陷的原因。