LGMD2I zebrafish characterization and drug screening

Dr. Louis Kunkel

Boston Children’s Hospital (Boston, MA)

 

Project Summary:

Zebrafish are good model systems to study human diseases and perform drug screening (cheaper and faster than mice). This grant will support the characterization and study of the FKRP zebrafish model. The zebrafish model containing FKRP mutations that mimic the human disease will be thoroughly characterized and will then be used to screen for existing drugs that could potentially improve muscle function in FKRP zebrafish. Drugs that show positive results in these models will then be tested in the FKRP mouse model in Dr. Qi Lu’s lab, at the Carolinas Medical Center.

This grant is jointly-funded by the LGMD2I Research Fund, the Samantha Brazzo Foundation and Cure CMD.