Parent Project Muscular Dystrophy to Fund $1 Million in Exon Skipping Projects

Leading Duchenne Organization Announces Request for Applications Focused on the Development of Oligos

HACKENSACK, N.J., Aug. 14, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) – the nation's leading voice for patients and families impacted by Duchenne muscular dystrophy (Duchenne) – announced that it will fund up to $1 million in projects by mid-2014 focused on the development of antisense oligonucleotides or other techniques to skip exons that are not currently in clinical development, or for multi-exon skipping projects, including the skipping of duplications. A Request for Applications (RFA) has just been released.

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Exon-skipping is a technique whereby out-of-frame deletions in a gene can be put back into frame by manipulating splicing patterns to strategically remove an up- or downstream exon in addition to the introns.  In Duchenne, the result is a shorter, but often still functional, dystrophin protein.  There are currently four companies around the world focused on developing exon-skipping strategies for Duchenne, with antisense oligonucleotides targeting exons 51, 44, 45 and 53 in clinical testing now. 

PPMD's Vice President of Research, Sharon Hesterlee, PhD, discusses the importance and timeliness of this kind of funding strategy: "As results for the leading exon 51 compounds continue to appear promising, the urgency has increased to expand the technique to correct less common deletions and to develop methods for skipping multiple exons that could also be applied to some duplications, as well as deletions. PPMD's mission is, and has always been, to help all people with Duchenne. We want to help maintain the momentum of this technology and encourage researchers to expand to additional exons and other mutations affecting our community."

Starting with small grants to the laboratories of Steve Wilton and Judith van Deutekom in 2003, PPMD has since invested over $600,000 in the development of exon-skipping as a treatment for Duchenne. 

PPMD will convene a special ad hoc committee to review all applications received.  Written reviews will be supplied to applicants and applicants will be allowed to respond one time to reviewer comments.  Based on applicant responses, final funding recommendations will be made by the ad hoc committee.  

This announcement is the latest focus of PPMD's broad research portfolio. Click here to learn more about this RFA and PPMD's Funding Portfolio or visit ParentProjectMD.org/Research.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.

SOURCE Parent Project Muscular Dystrophy



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