Drug Compound Screening for Dermatomyositis and Systemic Lupus Erythematosus

描述(申请人提供)：皮肌炎和系统性红斑狼疮是原因不明的自身免疫性疾病，没有特殊的药物治疗，通常使用有严重并发症的非特异性免疫抑制药物治疗。我们研究了皮肌炎和其他炎症性肌病患者的肌肉和血液样本，认为浆细胞样树突状细胞过度产生干扰素α和β(干扰素-a/b)是皮肌炎组织损伤的具体原因。 其他研究系统性红斑狼疮(SLE)的研究人员也假设，干扰素-a/b的过度产生是该疾病表现的部分原因。我们建议使用高通量药物发现方法来鉴定对糖尿病和系统性红斑狼疮具有预期疗效的化合物。将这些化合物开发成安全有效的治疗糖尿病和系统性红斑狼疮的药物是这项工作的长期目标。

DESCRIPTION (provided by applicant): Dermatomyositis and systemic lupus erythematosus are autoimmune disorders of uncertain cause and without specific medical therapy, generally treated with non-specific immunosuppressive medications that have significant complications. We have studied muscle and blood samples from patients with dermatomyositis and other inflammatory myopathies, and believe that overproduction of interferon alpha and beta (IFN-a/b) by plasmacytoid dendritic cells is responsible for tissue injury in dermatomyositis specifically. Other investigators studying systemic lupus erythematosus (SLE) have also hypothesized that such overproduction of IFN-a/b is in part responsible for the manifestations of that disease. We propose to use high-throughput drug discovery methods to identify compounds of predicted efficacy for DM and SLE. Development of such compounds into safe and effective drugs for the treatment of DM and SLE is the long-term objective of this work.