Myotonic Dystrophy Foundation Awards $200,000 for Research into Treatments for the Most Common Form of Adult-Onset Muscular Dystrophy

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Jan 23, 2014, 06:00 ET

MENLO PARK, Calif., Jan. 23, 2014 /PRNewswire-USNewswire/ -- The Myotonic Dystrophy Foundation (MDF) has awarded two $100,000 grants to postdoctoral fellows working in universities to encourage research in the management, treatment and cure of myotonic dystrophy (DM). Each of the 2014-2015 recipients will receive $50,000 a year for two years.

This award cycle brings MDF's total research funding to over $1.7M and builds on the Foundation's commitment to increasing the number of investigators focused on myotonic dystrophy research.

"The MDF fellows have created real impact in DM research since we started this program in 2009," says Molly White, executive director of the MDF. "Many have gone on to secure additional research funding from organizations such as the NIH, author important research publications, and help drive interest in DM research at major pharmaceutical companies. A major objective of the Foundation's work is advancing DM research and the fellows have been a significant part of how we achieve this objective."

The 2014-2015 awardees are:

Dr. Jintang Du, The Scripps Research Institute
Dr. Yao Yao, The Rockefeller University

Dr. Jintang Du
Dr. Du's research will seek to develop DNA-binding Py-Im polyamides (macromolecules with repeating units) to bind specific, identified CTG-CAG triplet repeats that cause DM1. Dr. Du and his lab partners have established that a specific Py-Im polyamide targeting CTG-CAG triplicate repeats is able to virtually abolish nuclear foci in patient cells. The molecule does not affect genes with short, non-pathogenic CTG/CAG repeats. Dr. Du will also investigate the pharmacokinetics, maximum tolerated dose and tissue distribution of the most effective molecules. Studies will be conducted on mouse models.

Dr. Yao Yao
Dr. Yao's research involves stem cells, and will seek to understand how pericytes (multipotent stem cells) can be used to treat DM. His research project will include investigating how laminin, which covers pericytes (stem cells) in normal conditions but degrades in disease conditions, affects specific functions of these stem cells. To this end, Dr. Yao will use a laminin-deficient mouse model he has developed, which shows severe muscular dystrophy soon after birth, similar to what is observed in congenital myotonic dystrophy. In addition to understanding the role of laminin on pericyte stem cell health and function, Dr. Yao will also investigate the mechanisms that drive laminin loss, and attempt to identify targets to control pericyte stem cell function and laminin loss in order to treat myotonic dystrophy and other muscular dystrophies.

About Myotonic Dystrophy: Described as "the most variable of all diseases found in medicine", myotonic dystrophy is an inherited disorder that can appear at any age and that manifests differently in each individual. The most common form of adult-onset muscular dystrophy, DM affects somewhere between 1:3,000 and 1:8,000 people worldwide, and can cause muscle weakness, atrophy and myotonia, as well as problems in the heart, brain, GI tract, endocrine, skeletal and respiratory systems. There is currently no treatment or cure for DM.

About the Myotonic Dystrophy Foundation: The Myotonic Dystrophy Foundation (MDF) is the world's largest DM patient organization. Its mission is Care and a Cure: to enhance the lives of people living with myotonic dystrophy, and advance research efforts focused on finding treatment and a cure for this disorder through education, advocacy and outreach.

Contact: Molly White
Myotonic Dystrophy Foundation
Phone: 866-968-6642
E-mail: [email protected]

SOURCE Myotonic Dystrophy Foundation