Posts by Thurman M. Wheeler, MD
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Novel Mouse Model Allows Rapid Evaluation of Candidate Drugs for Muscular Dystrophy
Neurologists at Massachusetts General Hospital have created a bitransgenic mouse model that expresses an alternative splicing reporter of myotonic dystrophy type 1 and enables rapid, noninvasive estimation of the pharmacodynamics of potential new drugs.
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Extracellular RNA in Urine Is a Source of Biomarkers for Muscular Dystrophy
Researchers at Massachusetts General Hospital are the first to show that mRNA splicing patterns in urine present a rich source of personalized biomarkers for muscular dystrophy—with implications for monitoring other systemic diseases.