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自身免疫性重症肌无力的当前治疗方法、新兴转化治疗方法和新的治疗靶点。

Current Treatment, Emerging Translational Therapies, and New Therapeutic Targets for Autoimmune Myasthenia Gravis.

机构信息

Neuromuscular Division, Department of Neurology, Duke University Medical Center, Durham, NC, USA.

Department of Neurological Sciences, Rush University Medical Center, Chicago, IL, USA.

出版信息

Neurotherapeutics. 2016 Jan;13(1):118-31. doi: 10.1007/s13311-015-0398-y.

Abstract

Myasthenia gravis (MG) is an autoimmune disease associated with the production of autoantibodies against 1) the skeletal muscle acetylcholine receptor; 2) muscle-specific kinase, a receptor tyrosine kinase critical for the maintenance of neuromuscular synapses; 3) low-density lipoprotein receptor-related protein 4, an important molecular binding partner for muscle-specific kinase; and 4) other muscle endplate proteins. In addition to the profile of autoantibodies, MG may be classified according the location of the affected muscles (ocular vs generalized), the age of symptom onset, and the nature of thymic pathology. Immunopathologic events leading to the production of autoantibodies differ in the various disease subtypes. Advances in our knowledge of the immunopathogenesis of the subtypes of MG will allow for directed utilization of the ever-growing repertoire of therapeutic agents that target distinct nodes in the immune pathway relevant to the initiation and maintenance of autoimmune disease. In this review, we examine the pathogenesis of MG subtypes, current treatment options, and emerging new treatments and therapeutic targets.

摘要

重症肌无力(MG)是一种自身免疫性疾病,与针对 1)骨骼肌乙酰胆碱受体;2)肌肉特异性激酶,一种对于维持神经肌肉突触至关重要的受体酪氨酸激酶;3)低密度脂蛋白受体相关蛋白 4,一种肌肉特异性激酶的重要分子结合伴侣;以及 4)其他肌肉终板蛋白的自身抗体的产生有关。除了自身抗体的特征外,MG 还可以根据受影响肌肉的位置(眼肌型与全身型)、症状发作的年龄以及胸腺瘤病理的性质进行分类。导致自身抗体产生的免疫病理事件在不同的疾病亚型中有所不同。我们对 MG 各亚型免疫发病机制的认识的进展将使我们能够有针对性地利用不断增加的治疗药物,这些药物针对与自身免疫性疾病的发生和维持相关的免疫途径中的不同节点。在这篇综述中,我们研究了 MG 各亚型的发病机制、当前的治疗选择以及新兴的新治疗方法和治疗靶点。

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