Matthew Alexander, a postdoctoral research fellow in genetics and pediatrics at Harvard Medical School in Boston, Mass., was awarded an MDA development grant totaling $180,000 over a period of three years to study the role of microRNAs in Duchenne muscular dystrophy (DMD).
MicroRNAs (miRNAs) are molecules that interact with genes, including the dystrophin gene. (Mutations in the dystrophin gene are the cause of DMD.) Those interactions help control a wide range of processes in cells.
When the dystrophin gene is mutated, the amount of a miRNA called miR-486 is greatly reduced. This causes changes in a system that regulates muscle growth and development.
“Currently, very little is known about the secondary signaling pathways that are altered as a consequence of dystrophin deficiency, and the roles that micro RNAs play in muscle disease,” Alexander says. His work will explore the effects of reducing miR-486 in the context of dystrophin deficiency in the mdx mouse, a research model of DMD. The goal is to gain a better understanding of the problems for muscles caused by loss of miR-486 in order to develop therapies to avoid or compensate for these problems.
“MicroRNA-based therapeutics, unlike traditional single-gene based drug treatments, are capable of manipulating the expression of entire signaling pathways,” says Alexander. In addition, “they can be delivered in a dose-dependent fashion, and can reach the damaged muscle via intravascular or intramuscular injections,” all potential advantages for developing effective treatment.
Funding for this MDA grant began Feb. 1, 2013.