MDA awarded a research grant totaling $253,812 over two years to Dwight Koeberl, associate professor in the department of pediatrics at Duke University Medical Center in Durham, N.C. The funds will help support Koeberl’s research on the development of gene therapy for acid maltase deficiency (AMD, or Pompe disease).
Koeberl is investigating a new therapy for Pompe disease, using a drug that improves the response to gene therapy. The drug, called a beta2-agonist, increases the amount of acid maltase (the enzyme that’s deficient in Pompe disease) taken up by muscle cells during gene therapy.
Koeberl and colleagues also are studying ways to stop the immune system from mounting a response and interfering with gene therapy.
Experiments are being conducted in a research mouse model of Pompe disease. The mouse, which lacks the gene for acid maltase, responds to therapy in a similar way to people with Pompe disease.
Favorable results in Koeberl’s study could lead to a new design for gene therapy in Pompe disease.
Funding for this MDA grant began Aug. 1, 2012.