Charles Thornton, Ph.D.

Dr. Thornton's research program studies myotonic dystrophy, a common form of muscular dystrophy that begins in adulthood and is characterized by progressive muscle wasting and weakness. For this project, he focuses on myotonic dystrophy type 1 (DM1) which results from an unconventional RNA mutation. To attack DM1 at its root cause, he has partnered with industry colleagues to define the therapeutic properties of newly-developed reagents called antisense oligonucleotides (ASO) that target the toxic RNA in DM1. Expanding the understanding of these reagents will inform and guide their use in therapeutic trials and in clinical practice.