Development of Soluble mini-Agrin as a Treatment for Dystroglycanopathies

Dr. Jasbir Seehra

Achelois Biosciences (Lexington, MA, USA)

 

Project summary:

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.