Duchenne muscular dystrophy research received a boost from Parent Project Muscular Dystrophy (PPMD) in the form of a $250,000 grant awarded to Dr. Eric Olson and the Department of Molecular Biology at the University of Texas Southwestern Medical Center.
Olson will use the grant specifically to continue research on CRISPR/Cas9 technology to find a cure for Duchenne, which affects one in every 5,000 live male births making it the most common fatal genetic disorder.
"Thank you to Pat Furlong and the PPMD community for supporting this exciting technological advance in the treatment of Duchenne muscular dystrophy,” Olson said. “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.”
Dystrophin proteins in CRISPR/Cas9 in mice will continue to be studied in order to compare them to proteins from other therapeutic approaches. Olson also wants to make sure the CRISPR targets the exact genetic code and does not produce any ‘off-target’ effects.
"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,” Abby Bronson, PPMD's senior vice president of research strategy, said. “This in-depth look at safety will help us more comprehensively identify any risks associated with CRISPR/Cas9."
