Department of Neurology, Kumamoto University, Kumamoto, Japan
Department of Neurology, International University of Health and Welfare Narita Hospital, Narita, Japan.
J Neurol Neurosurg Psychiatry. 2023 Dec;94(12):1018-1024. doi: 10.1136/jnnp-2023-331474. Epub 2023 Jul 14.
Sporadic inclusion body myositis (sIBM) is an intractable muscle disease that frequently affects elderly people. Autoantibodies recognising cytosolic 5'-nucleotidase 1A (cN1A) were found in the sera of patients with sIBM. However, the pathogenic role of the autoantibodies remained unknown. This study investigated the pathogenic properties of the autoantibodies using active cN1A peptides immunisation.
Wild-type C57BL6 mice were injected with three different mouse cN1A peptides corresponding to the previously reported epitope sequences of human cN1A. After confirming the production of autoantibodies to the corresponding cN1A peptides in each group, changes in body weight, exercise capacity by treadmill test and histological changes in mice injected with cN1A peptides or controls were investigated.
Autoantibodies against cN1A were detected in serum samples from mice injected with cN1A peptide. Some groups of mice injected with cN1A peptide showed significant weight loss and decreased motor activity. The number of myofibres with internal nuclei increased in all the peptide-injected groups, with surrounding or invading CD8-positive T cells into myofibres, abnormal protein aggregates and overexpression of p62 and LC3.
Active cN1A peptide immunisation partially reproduced the clinical and histological aspects of sIBM in wild-type mice. The murine model demonstrates the pathogenic properties of anti-cN1A autoantibodies to cause sIBM-like histological changes.
散发性包涵体肌炎(sIBM)是一种常见于老年人的难治性肌肉疾病。在 sIBM 患者的血清中发现了识别细胞质 5'-核苷酸酶 1A(cN1A)的自身抗体。然而,这些自身抗体的致病作用尚不清楚。本研究使用活性 cN1A 肽免疫来研究自身抗体的致病特性。
野生型 C57BL6 小鼠用三种不同的小鼠 cN1A 肽进行注射,这些肽对应于先前报道的人 cN1A 的表位序列。在确认每组中针对相应的 cN1A 肽产生自身抗体后,研究了注射 cN1A 肽或对照的小鼠的体重变化、跑步机测试的运动能力以及组织学变化。
在注射 cN1A 肽的小鼠血清样本中检测到针对 cN1A 的自身抗体。一些注射 cN1A 肽的小鼠组表现出明显的体重减轻和运动能力下降。所有肽注射组的肌纤维内核数量增加,伴有 CD8 阳性 T 细胞浸润到肌纤维中、异常蛋白聚集和 p62 和 LC3 的过度表达。
活性 cN1A 肽免疫部分复制了野生型小鼠中 sIBM 的临床和组织学特征。该小鼠模型证明了抗 cN1A 自身抗体的致病特性,可导致类似于 sIBM 的组织学变化。