Dr. Jasbir Seehra
Achelois Biosciences (Lexington, MA, USA)
This project will test a novel recombinant protein, called Mini-Agrin, as a treatment for dystroglycanopathies. The project is split into 2 phases: the initial phase will test whether mini-agrin can overcome the defect in alpha-dystroglycan caused by FKRP mutations using cell lines. If this initial phase is successful, the researchers will test its use to treat the mouse model of FKRP mutation.