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The role of miRNAs in ALS and their use as a biomarker of disease progression
(Collaborators: Eran Hornstein and Adriano Chio’)
Aim: to comprehensively address the potential of plasma miRNA as disease biomarkers, to further use mouse models to gain an understanding of the biological processes that determine plasma miRNA signatures, and to investigate whether a pharmacological intervention affecting miRNA processing can impact the course of ALS.
The potential of serum/plasma miRNAs as an ALS biomarker has been highlighted by several studies. Unlike mRNAs, miRNAs are stable in serum making them an accessible and reliable measurement in patients.
In contrast to other canonical biomarkers (e.g. serum proteins) that each require a specific assay, thousands of miRNA transcripts can be probed with one single assay, allowing the identification of multiple complex signatures, which can be integrated in a multi-modal analysis with other biomarkers, clinical data and imaging, to identify disease subgroups. Several studies have investigated serum miRNAs as potential biomarkers in ALS and have identified changes (De Felice et al., 2014; Freischmidt et al., 2013, 2014; Toivonen et al., 2014).
We use miRNAseq to investigate the levels of serum/plasma miRNA, and apply this to a very well curated longitudinal collection opf patient samples and a range of mouse models.