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.