Grant Provides Funding Toward Gene Therapy Clinical Trial to Replace the DMD Gene in All of the Leg Muscles of Duchenne Patients

February 15, 2012

Investigators in the Center for Gene Therapy in The Research Institute at Nationwide Children’s Hospital are working toward an approach to replace the defective gene in Duchene muscular dystrophy (DMD) by treating all of the muscles in the leg, thanks to funding from Jesse’s Journey.

Duchenne, the most common form of childhood muscular dystrophy, is a progressive and fatal muscle disorder affecting boys and young men that causes the loss of muscle function.  Duchenne muscular dystrophy is caused by a defective gene for dystrophin, a protein in the muscles. Investigators in the Center for Gene Therapy have been working to replace the missing or defective dystrophin gene by using adeno-associated virus (AAV) as a vehicle for gene transfer.

To continue this work, Jesse’s Journey Foundation for Gene and Cell Therapy has awarded the team with a three-year, $300,000 grant. The funding will allow investigators to develop an approach to treat all of the muscles of the leg, using preclinical models of DMD. They will use AAV as a vehicle to deliver micro-dystrophin to leg muscles through arterial circulation.  

“In this approach all of the muscles of the leg will be treated increasing the potential for significant improvement in the quality of life,” said Louise Rodino-Klapac, PhD and Louis Chicoine, MD who are co-principal investigators for the grant. The Center for Gene Therapy Director Jerry Mendell, MD, is also a co-investigator for the study and will help pave the way to the clinic. 

About Nationwide Children's Hospital

Named to the Top 10 Honor Roll on U.S. News & World Report ‘s 2018-19 list of “Best Children’s Hospitals,” Nationwide Children’s Hospital is one of America’s largest not-for-profit freestanding pediatric health care systems providing wellness, preventive, diagnostic, treatment and rehabilitative care for infants, children and adolescents, as well as adult patients with congenital disease. Nationwide Children’s has a staff of more than 13,000 providing state-of-the-art pediatric care during more than 1.4 million patient visits annually. As home to the Department of Pediatrics of The Ohio State University College of Medicine, Nationwide Children’s physicians train the next generation of pediatricians and pediatric specialists. The Research Institute at Nationwide Children’s Hospital is one of the Top 10 National Institutes of Health-funded freestanding pediatric research facilities. More information is available at NationwideChildrens.org.