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.

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