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Principal Investigator: Dr. Kevin P. Campbell
Dr. Campbell is using mouse models to explore the mechanistic causes and therapeutic potential of improving muscle membrane maintenance and repair for the treatment of dystroglycan-related muscular dystrophies.
This project is exploring our basic understanding of how dystroglycan binds laminin, and the effectiveness of expressing LARGE and applying drugs of several classes in treating dystroglycan-related muscular dystrophies. We are investigating the role of a novel post-translational phosphate modification -- an O-linked mannose that is part of the laminin-binding glycan structure of alpha-dystroglycan -- in laminin binding. These studues will improve our understanding of the modifications required for dystroglycan to serve as a laminin receptor, and advance patient diagnosis by improving biomarker correlations with clinical severity. This research will provide a platform for new therapeutic avenues, some of which will be possible to implement directly in the care of dystroglycanopathy patients.
Dr. Campbell's Research Laboratory Website
Dr. Campbell's Department of Molecular Physiology & Biophysics Website
Dr. Campbell's HHMI Investigator Website
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