Extracellular RNA Biomarkers of Duchenne Muscular Dystrophy
Abstract
Scientific objective: Our goal is to use urine and/or blood samples from Duchenne patients to identify markers of muscle cell dysfunction that can be used to determine whether new treatments are working. Rationale: Small particles called extracellular vesicles (EVs) are released from many different cell types into the urine and blood. EVs contain molecules called extracellular RNAs (exRNAs). In urine and blood, EVs contain exRNA biomarkers of cancers and other disease states. Recently, our group was the first to demonstrate that EVs in urine also contain a certain type of exRNA that can serve as specific biomarkers of Duchenne and other muscular dystrophies (Antoury, et al., 2018; Antoury, et al., 2019). Our data suggest that exRNAs are a rich and renewable biomarker source for Duchenne muscular dystrophy that could enable monitoring of the disease state and the molecular response to a new treatment. Focus Area: Assessment of clinical trial tools and outcome measures, specifically the discovery and qualification of pharmacodynamic and predictive biomarkers.
Document Details
- Document Type
- Technical Report
- Publication Date
- Aug 01, 2020
- Accession Number
- AD1118341
Entities
People
- Thurman Wheeler