Approaches to therapy for the skeletal muscle actin diseases [ 2012 - 2014 ]

Research Grant

Researchers: Prof Nigel Laing (Principal investigator) ,  A/Pr Nigel Clarke ,  Dr Kristen Nowak ,  Prof Kathryn North

Brief description We have shown that errors in a crucial muscle protein called actin cause muscle diseases that affect newborn children. These diseases are mainly very severe, causing death within the first year of life. Currently there is no cure. This project will investigate possible therapies for these diseases, such as viral delivery of a normal version of actin and finding a drug to overcome the weakness. Successful outcomes will crucially bring treatment closer for the patients.

Funding Amount $AUD 912,078.47

Funding Scheme Project Grants

Notes Standard Project Grant