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Finding the Right CRISPR Targets for DM

CRISPR is an exploratory strategy with potential for treatment of RNA-triggered diseases. Will DNA or RNA targeting prove to be the best approach for DM?

IL-6 Myokine Signaling in CDM

Genetic and epigenetic mechanisms drive differences between CDM and DM1—a new study elucidates downstream signaling pathways that underlie their diverse phenotypes and represent putative therapy development targets for CDM.

Interaction of Age and Gender in DM2

Age and gender impact the onset and progression of DM2, but the pattern shows both similarities and differences from that of DM1.

What We Do (and Don’t) Know About Cognitive Function in DM1

A new literature review suggests meta-analysis of cognitive studies in DM1 is both feasible and can inform understanding of disease mechanisms and patient management.

The Origin of Insulin Resistance in DM

It would be easy to conclude that insulin resistance and diabetes in myotonic dystrophy is a product of mis-splicing of INSR transcripts—but is that the full story?

Made to Measure: Developing Clinical Tools to Capture the Severity and Progress of DM

Learn more about Dr. Ami Mankodi, principal investigator at the National Institutes of Health’s (NIH) National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda, Maryland. Dr. Mankodi has been involved in research that has helped shape a fundamental biologic and molecular understanding of myotonic dystrophy (DM).

Modifying Gene Editing Technology for DM

Investigators at the University of California San Diego, the University of Florida, and the National University of Singapore have recently reported early research that potentially ‘repurposes’ gene editing technology for a set of RNA disorders—myotonic dystrophy type 1 (DM1), myotonic dystrophy type 2 (DM2), a subset of Lou Gehrig’s disease (ALS) patients and Huntington’s disease. They have modified the Cas9 enzyme so it is targeted to toxic RNA, instead of the expanded DNA repeats in these diseases.

Know Your DM1 Repeat Length: It’s Important for Your Cardiac Care

Understanding cardiac and other DM risk factors and planning for the known complications of DM that may affect you can help protect and maintain your quality of life and that of your loved ones.

Molecular Events Underlying Congenital DM

A new study points to the timing of MBNL-dependent RNA processing defects as a major factor in the pathogenesis of CDM.

Understanding and Measuring Fatigue in DM1

Although fatigue represents a substantial burden in DM1, tools must be validated to assess its diverse contributing factors in order to develop clinical trial endpoints and effective therapies.

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