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Applying Gene Editing Technology to RNA Diseases

For the first time, researchers have a means of targeting RNA in living cells. There is potential for this approach to edit the CTG and CTTG expansions in DM1 and DM2. Read the article from MDF to learn more.

Rare Chemistry: Matt Disney Advances Development of Small Molecule Therapeutics for DM

Dr. Matt Disney brings an unusual and increasingly valuable skill to therapy development for DM—he’s a chemist. The NIH has recently awarded two research grants in support of Dr. Disney’s research. Read this article to find out how his work leverages the latest advances in RNA biology to target the unique disease mechanisms of DM.

Gender Matters in DM1

Dr. Guillaume Bassez and a large team in France and Canada recently published an analysis of gender and its impact on the DM1 phenotype. Read about their findings in this article.

Molecular Changes Lead to Cardiac Dysfunction in DM

A new study by a multi-disciplinary, international team has shed light on the molecular changes at the root of DM-related heart dysfunction, the second leading cause of death for DM patients.

The Genetics of DM1 Repeat Size

Researchers from the University of Costa Rica and the University of Glasgow teamed up to investigate the DNA mutation causing myotonic dystrophy type 1 (DM1). They found that polymorphism in the MSH3 mismatch repair gene is associated with the levels of somatic instability of the expanded CTG repeat in the blood DNA of myotonic dystrophy type 1 patients. 

MDF Research Fellow Profile: Dr. Melissa Dixon

Dr. Dixon at the University of Utah has been awarded a 2016-2017 postdoctoral fellowship for her research applying fMRI to evaluate connectivity networks in the brains of children with congenital-onset myotonic dystrophy (CDM). Read more here about the work of Dr. Dixon and her colleagues.

Dr. Charles Thornton Wins Prestigious Javits Award

Dr. Charles Thornton, neurologist at the University of Rochester Medical Center and MDF Scientific Advisory Committee member, has been awarded a Javits Neuroscience Investigator Award from the National Institutes of Health to further his research on muscular dystrophy. Read more about this very distinguished award for “exceptional” investigators and the important work Dr. Thornton is doing here.

University of Iowa Launches Brain Imaging Study

The University of Iowa has been awarded a grant by the National Institute of Neurological Disorders and Stroke (NINDS) to fund a 3-year longitudinal study of adults with a family history of DM1. Learn more about the study and how to participate in this article.

 

Research Fellow Profile: Dr. Ian DeVolder

MDF has awarded a 2016-2017 postdoctoral fellowship to Dr. Ian DeVolder at the University of Iowa to study CNS-related impacts of DM1.

University of Florida - Sanofi Collaboration Receives DM Drug Screening Grant

In a ground-breaking academic and pharmaceutical company collaboration, Sanofi-Aventis and the University of Florida will screen for new drugs to treat DM1 and DM2. Read the full article here.

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