Parent Project Muscular Dystrophy Awards UT Southwestern Medical Center $250,000 Grant to Explore CRISPR/Cas9 Technology in Duchenne Muscular Dystrophy

HACKENSACK, N.J., Jan. 30, 2017 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced a $250,000 grant to be awarded to Dr. Eric Olson and the Department of Molecular Biology at UT Southwestern Medical Center. This grant, part of PPMD's Gene Transfer Initiative, will support Dr. Olson's ongoing study of CRISPR/Cas9 technology as a potential treatment for Duchenne.

Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately one in every 5,000 live male births.

This grant will help Dr. Olson and his team answer questions regarding the potential effectiveness and safety of CRISPR/Cas9 using animal models with Duchenne. "Thank you to Pat Furlong and the PPMD community for supporting this exciting technological advance in the treatment of Duchenne muscular dystrophy. We are optimistic about the potential of CRISPR/Cas9 to help Duchenne patients. There is still work to do, and this grant will help maintain the momentum of our research activities and for that we are grateful," said Dr. Olson.

Dr. Olson's lab will study the various types of dystrophin proteins produced using CRISPR/Cas9 in mouse models. In addition, they will compare the dystrophin proteins produced from CRISPR/Cas9 to proteins produced from other therapeutic approaches, such as the microdystrophins being researched for use in gene transfer.

Dr. Olson's lab will also investigate whether there are any unwanted "off-target" effects.  CRISPR/Cas9 works as a pointer through its "RNA guidewires," directing the CRISPR to the exact area of the genetic code where it needs to go. What remains unknown is how often the system finds an area that is close but not exact, yet still ends up cutting the genetic code there. This unintentional cut could lead to what is considered "off target" effects.

Abby Bronson, PPMD's Senior Vice President of Research Strategy explains, "Because CRISPR/Cas9 is new and never been used in humans, the safety of this technology will be thoroughly examined, including looking for any immune response to the AAV9. This in-depth look at safety will help us more comprehensively identify any risks associated with CRISPR/Cas9."

For a recent webinar with Dr. Olson discussing next steps with this project, click here.

PPMD has a long history of supporting early-stage, innovative research, providing funding at a critical moment in a therapy's development. Gene transfer has been explored for years as a possible therapeutic approach to treating Duchenne. Only recently, though, have advances in science and technology made it seem like a viable treatment option for Duchenne, with CRISPR/Cas9 recently making headlines as a potential therapy for a variety of disorders.

According to PPMD founding President and CEO, Pat Furlong, funding for this grant came together as a result of the organization's extremely successful 2016 year-end campaign: "Every donation, from $5 to $50,000 is important in the fight to end Duchenne. We were overwhelmed with the generosity and support of this community, once again. As a result of our fundraising efforts, not only will PPMD be able to support Dr. Olson's lab as they take a deeper dive into the potential of CRISPR/Cas9 technology as a potential treatment for Duchenne, but we will be able to look at other CRISPR/Cas9 studies being conducted. Extensive and thorough exploration of innovative technology like CRISPR/Cas9 and gene therapy takes time, something people with Duchenne have precious little. But our organization's ability to provide grants to researchers like Dr. Olson, we believe, will shorten the amount of time this research takes. We are humbled by the Duchenne community's investment in PPMD and our innovative research approach."

The $250,000 grant to Dr. Olson's lab and UT Southwestern Medical Center was made possible in part by donations from individuals and families, as well as gifts from Duchenne foundations, including: The Ackerman Foundation and Nicholoff Family, Hope for Gabe, Hope for Gus, John Owen's Adventure, Kindness Over Muscular Dystrophy (including the Curran & Leahy families), Little Hercules Foundation, Ryan's Hope for a Cure Charitable Foundation, and the Zack Heger Foundation. Special thanks to the Killian family for providing a matching gift during this campaign. PPMD is grateful to all of these foundations, families, and every individual who helped PPMD fund this project. We appreciate your partnership.

Earlier this month, PPMD announced a $2.2 million grant to Dr. Jerry Mendell, Dr. Louise Rodino-Klapac, and Nationwide Children's Hospital to explore gene therapy as a potential treatment for Duchenne. "PPMD believes in collaborating across stakeholder groups – patients, caregivers, researchers, clinicians, companies, and regulators – working together to move exciting new research strategies forward. We believe this is the moment to commit to the incredible research taking place at UT Southwestern Medical Center and Nationwide Children's Hospital, as part of our larger Gene Transfer Initiative which we look forward to rolling out in the coming weeks," says Furlong.

To learn more about PPMD's research investments, please visit the website.

About Parent Project Muscular Dystrophy

Duchenne is a fatal genetic disorder that slowly robs people 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 people 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 people with Duchenne live longer, stronger lives. We will not rest until every person 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. Follow PPMD on Facebook, Twitter, and YouTube.

SOURCE Parent Project Muscular Dystrophy

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