Juvenile Myositis

青少年肌炎

• 批准号: 10683016
• 负责人: James Inglese
• 金额: $ 23.14万
• 依托单位: NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES
• 依托单位国家: 美国
• 资助国家: 美国
• 起止时间: 至
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10683016
• 关键词: Adolescent; Affect; Autoimmune Diseases; Biological Assay; Calcinosis; Cells; Child; Collaborations; Contracture; Data Analyses; Dermatomyositis; Disease; Environmental Risk Factor; Exanthema; Financial Support; Foundations; Gastrointestinal tract structure; Genetic; Goals; Immune system; Libraries; Life; Muscle; Muscle Weakness; Myositis; Pathogenesis; Pathway interactions; Pharmaceutical Preparations; Polymyositis; Skin; Symptoms; Testing; Tissues; Toxin; Ulcer; Work; assay development; base; design; environmental agent; experience; functional genomics; novel; small molecule libraries; therapy development

Juvenile myositis (JM) is an autoimmune disease targeting muscles, skin and other tissues caused by yet to be determined genetic and environmental factors. With financial support from the Cure JM Foundation in collaboration with Cure JM Foundation scientific advisors we have completed the design and development of assays based on genetic anomalies that may be contributing to JM pathogenesis. These assays have been used in the interrogation of chemical libraries for the identification of molecules that modify these aberrant pathways. We have completed the testing of an approved drug library revealing known and novel leads for JM therapy development. Additionally, a library of toxins and environmental agents that may be involved disease initiation or exacerbation has been evaluated, and analysis of this data is ongoing.

青少年肌炎（JM）是一种以肌肉、皮肤和其他组织为靶点的自身免疫性疾病，由尚未确定的遗传和环境因素引起。在Cure JM基金会与Cure JM基金会科学顾问的合作下，我们已经完成了基于可能导致JM发病机制的遗传异常的检测的设计和开发。这些试验已用于化学文库的审讯，以确定修饰这些异常途径的分子。我们已经完成了一个被批准的药物库的测试，揭示了JM治疗开发的已知和新的线索。此外，还对可能引起疾病发生或恶化的毒素和环境因子库进行了评估，并正在对这些数据进行分析。