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The development of medical approaches requires preclinical and clinical trials for evaluation of therapeutic efficacy. Such analysis entails the usage of biomarkers, which offer 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 extended to plasma samples, suggesting that they could also be of curiosity as biomarkers. First, we demonstrated that dystromiRs dysregulation occurs in MD with both preserved or disrupted expression of the dystrophin-associated glycoprotein complicated, supporting the utilization of dystromiRs as generic biomarkers in MD. Then, we geared toward evaluation of the capability of miRNAs as monitoring biomarkers for experimental therapeutic approach in MD. To this finish, we took advantage of our previously characterized gene therapy approach in a mouse model for α-sarcoglycanopathy. We identified a dose-response correlation between the expression of miRNAs on both muscle tissue and blood serum and the therapeutic profit as evaluated by a set of new and classically-used evaluation methods.
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