MicroRNA targeting to achieve Systemic Sclerosis C.. (MICROSCAP)
MicroRNA targeting to achieve Systemic Sclerosis Control and Prediction
Start date: Apr 1, 2014,
End date: Mar 31, 2016
Systemic Sclerosis (SSc) is a disfiguring disease with high mortality and morbidity, characterized by progressive and uncontrollable fibrosis of the skin and internal organs. There is no successful therapy for SSc resulting in a markedly shortened life expectancy, usually less that most cancers. Moreover, SSc diagnosis is very difficult in the early phase of the disease; consequently patient treatment is delayed until skin and/or internal organ involvement is evident and already irreversible. The pathology, therefore, presents an extreme need of both effective therapies and early diagnosis/prognosis markers, in order to treat SSc patients before the development of severe organ complications. The proposed project builds on the unique observation that plasmacytoid dendritic cells (pDC) are dysregulated in the early disease onset of SSc, due to an aberrant microRNA (miRNA) expression pattern. The aim of the proposal is to delineate the critical role of identified miRNAs in pDC dysregulation and translate it to clinical relevant instruments to prevent or treat SSc. Thanks to my scientific and technical expertise in the study of miRNAs and immune regulation, I will be able to dissect the aberrant mechanisms leading to pDC dysregulation via miRNA-regulated pathways. This will provide the basis to develop novel therapeutic approaches to dampen the immune alterations present in SSc and control the disease progression. Furthermore, the analysis of a unique cohort of preclinical SSc cohort present in Radstake’s group and its expertise in SSc field will allow me to identify miRNA profiles that predict the progression of early-SSc into definite SSc. The marriage between my own and Radstake’s group expertise will therefore create the perfect ground to open novel perspectives for a more effective treatment and prevention of SSc, reducing the disease impact on health and the economic resources employed for SSc patients, with an overall advantage for the entire European Community.
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