Djupare förståelse av miRNA i modellsjukdomar för autoimmunitet och neuromuskulär transmission

Tidsperiod: 2015-01-01 till 2017-12-31

Projektledare: Anna Rostedt Punga

Finansiär: Vetenskapsrådet

Bidragstyp: Projektbidrag

Budget: 3 000 000 SEK

Patients with the autoimmune neuromuscular disorder Myasthenia Gravis (MG) suffer from abnormal muscle fatigue and weakness due to antibody attack of the neuromuscular synapse. MG is considered a model disease of autoimmunity in the peripheral nervous system. To date there are no available biomarkers in autoimmune neuromuscular disorder, including MG, that correlate with disease severity or progress. Abnormal accumulation of small non-coding microRNAs (miRNAs) correlates with the pathogenesis of diseases and the quantitative detection of miRNAs has been considered a novel blood-based biomarker tool to define disease status. Nevertheless, no studies have investigated the role of miRNAs in the pathogenesis or disease course of neuromuscular autoimmune disorders. This study will elucidate miRNA profile in MG patients and establish a correlation between miRNA profile and disease course and status and specific miRNAs will be modified in peripheral blood monocyte cells. Further, the expression of miRNAs will be analysed in muscles from mice with experimental autoimmune MG and the effect of dysregulated miRNAs will be assessed at the neuromuscular junction in-vitro. As biomarkers are lacking, miRNA profiling will provide an essential complement to allow close monitoring of the disease course. The results will contribute to better understanding of the role of miRNA in the disease process of autoimmune neuromuscular disorders, where MG is considered a model disease.