In this presentation you will learn about many of the key scientific discoveries that have allowed researchers, clinicians and the biotechnology and pharmaceutical industries to develop therapeutic strategies for myotonic dystrophy. You will learn about the identification of the mutations in the genes that cause DM1 and DM2 and how understanding the mechanism through which these mutations cause DM. You will learn how the mutations can change over time in a person and why individuals with DM have such a wide range of symptoms. This foundation of DM knowledge has provided researchers with diverse ways to approach DM therapies and treatments. Dr. Berglund is introduced by Support Group Facilitator Suzanne Perkins.
Presented during the Understanding DM Research and Progress Track at the MDF 2020 Virtual Conference.
About the presenter:
Andrew Berglund, PhD
Dr. Berglund's scientific career has revolved around understanding the role of RNA in biology. The focus of his lab is neuromuscular diseases with the goal of translating basic science into therapeutic strategies using a combination of biochemical, cellular, genomic and computational approaches. Research in the Berglund Lab has focused on RNA splicing with an emphasis on understanding the mechanisms of a common form of muscular dystrophy, myotonic dystrophy, as well as other microsatellite diseases such as amyotrophic lateral sclerosis (ALS), and spinocerebellar ataxias (SCAs). Learn more about the work of the Berglund Lab at The RNA Institute.