Thomas Cooper, professor of pathology and immunology at Baylor College of Medicine in Houston, Texas, was awarded an MDA research grant totaling $300,000 over a period of three years to develop “antisense” therapy approaches to treat type 1 myotonic muscular dystrophy (MMD1, also known as DM1).
MMD1 is caused by an expanded section of a gene, which in turn causes excess accumulation of a normal cellular molecule called RNA. The extra RNA then traps other molecules that are important for processing the genetic instructions that are used to make proteins. Cooper has created a mouse model of MMD1 in which he has begun to explore the details of these effects, and in which he will now begin to test a therapeutic strategy for the disease. He is developing antisense oligonucleotides — short RNA-like molecules that bind to the excess RNA, blocking its interactions with other substances or causing it to be broken down. A particular challenge is delivering the antisense oligonucleotides to the heart muscle, which is affected in MMD1; Cooper will be exploring how best to accomplish this in the mouse.
“There has been rapid progress in the use of antisense molecules as a potential therapy for myotonic dystrophy,” Cooper says. "In animal models, there has been some success delivering the therapy to skeletal muscles. We will focus on delivery of the antisense oligonucleotides to the heart.”
Funding for this MDA grant began August 1, 2013.