UCLA scientists are studying ways to correct genetic mutations that cause Duchenne muscular dystrophy.
Scientists at the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research and Center for Duchenne Muscular Dystrophy at UCLA have received funding from California’s stem cell agency for the next phase of cutting-edge stem cell gene therapy research that could eventually produce a treatment for Duchenne muscular dystrophy.
Duchenne, which affects approximately 1 in 5,000 boys in the U.S., is the most common fatal childhood genetic disease.
The two-year, $2.15 million grant from the California Institute for Regenerative Medicine will fund research by UCLA’s April Pyle, Melissa Spencer and Huan Meng.
The scientists will utilize a stem cell gene editing technology called CRISPR/Cas9 in two different approaches to correct the genetic mutations that cause Duchenne. The two techniques will use nanoscale particles that will be tested in mice to determine if the methods could be used in a clinical setting in the future.
“We are excited to use the latest high-tech approaches to further our research and hopefully someday create a treatment for Duchenne,” said Spencer, a professor of neurology at the UCLA David Geffen School of Medicine, co-director of the Center for Duchenne Muscular Dystrophy and a member of the Broad Stem Cell Research Center. “We’re thankful that the California Institute for Regenerative Medicine has funded our work.”
The CRISPR/Cas9 platform for Duchenne developed at UCLA is not yet available in clinical trials and has not been approved by the FDA for use in humans.