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The development of medical approaches requires preclinical and clinical trials for evaluation of therapeutic efficacy. Such analysis entails using biomarkers, which give data on the response to the therapeutic intervention. One newly-proposed class of biomarkers is the microRNA (miRNA) molecules. In muscular dystrophies (MD), the dysregulation of miRNAs was initially noticed in muscle biopsy and later prolonged to plasma samples, suggesting that they could also be of interest as biomarkers. First, we demonstrated that dystromiRs dysregulation occurs in MD with either preserved or disrupted expression of the dystrophin-related glycoprotein advanced, supporting the utilization of dystromiRs as generic biomarkers in MD. Then, we aimed toward evaluation of the capacity of miRNAs as monitoring biomarkers for experimental therapeutic method in MD. To this finish, we took benefit of our beforehand characterized gene therapy strategy in a mouse mannequin for α-sarcoglycanopathy. We identified a dose-response correlation between the expression of miRNAs on each muscle tissue and blood serum and the therapeutic benefit as evaluated by a set of latest and classically-used analysis strategies.
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發表於 2025-8-12 05:46:03