Dr. James Dowling
University of Michigan (Ann Arbor, MI)
This grant will support the development and characterization of a stable FKRP zebrafish model. After characterization, the model will be used for screening of potential drug candidates. Another zebrafish model of dystroglycanopathy (Pomt2 deficiency) will be tested in parallel to the FKRP model, for comparison. This will bring further insights into the disease process among the alpha-dystroglycan-related diseases. In order to potentially expand the impact of the discoveries made in this project, all results will be shared through an open-source database.
This grant is jointly-funded by the Samantha Brazzo Foundation, the LGMD2I Research Fund and Cure CMD.