Gersbach Earns Prestigious Hartwell Award

Charles A. Gersbach, PhD, a researcher in the Pratt School of Engineering’s Department of Biomedical Engineering, has been selected to receive a Hartwell Foundation Individual Biomedical Research Award for his work to correct faulty genes that cause Duchenne muscular dystrophy (DMD).

He will receive support of $100,000 per year for three years. Gersbach is one of only 10 individuals from different institutions to receive one of the prestigious awards.

Duchenne muscular dystrophy is an inherited disorder that involves rapidly-worsening muscle weakness. Males are more susceptible than females. It affects one in every 3,600 infants. Symptoms usually appear before age 6, and by age 12, most sufferers are confined to a wheelchair.

Gersbach’s lab is working on technology to edit the genes responsible for making proteins–– quite literally, to correct the abnormal DNA sequence in the DMD gene.

“We’re building these proteins—or enzymes—to go in, recognize and combine with the particular (Duchenne) gene among the 3 billion genes in the human body,” he says. “We have seen promising preliminary results that indicate that the enzymes we have designed can do this. Now we need to prove it in the animal context.”

"(Gersbach) is pursuing a conceptually and technically risky idea with a big benefit to children if he is successful," said Hartwell Foundation President Frederick Dombrose, Ph.D.

Gersbach uses the example of car headlights to compare his gene editing technique to conventional gene therapy. If one headlight burns out, conventional gene therapy simply adds another headlight. "But you have no say in which direction the new headlight faces or how effective it will be," he says. His process actually replaces the damaged headlight.

Gersbach will use his hand-crafted enzymes to correct the Duchenne mutation in animal cells in tissue culture, and subsequently inject them into a mouse affected with DMD.

“This is where the Hartwell Foundation Award is so vital,” Gersbach says, “because the foundation is committed to funding early, yet promising research that has not yet qualified for significant funding from outside sources.”

Hartwell Foundation President Frederick Dombrose, Ph.D., says Charles Gersbach is extraordinarily innovative. “He is the prototypical researcher we seek to fund, in that he is pursuing a conceptually and technically risky idea with a big benefit to children if he is successful.”

Duke was one of 10 institutions nationwide that qualified to compete for the foundation’s Individual Biomedical Research Awards. These selected institutions held their own internal competitions to nominate four principal investigators for individual recognition.

“This award highlights the creativity and innovation of our faculty in biomedical science and engineering,” said Duke University President Richard H. Brodhead. “We are pleased to have The Hartwell Foundation’s partnership to help develop early-stage research with the potential to foster broad advances in medicine to benefit children.”

Duke Chancellor for Health Affairs Victor J. Dzau, MD, said, “Partnerships with private foundations such as The Hartwell Foundation are increasingly important in academic medicine and the Hartwell award is exciting because of its potential to speed the translation of basic science into new therapies.”

The Hartwell Foundation began in 1999, and its initial gift was to the St. Jude Children’s Research Hospital in Memphis to establish the Hartwell Center, a unique biomedical resource for integrating high throughput biotechnology and bioinformatics with academic programs.

In selecting participating institutions like Duke University, The Hartwell Foundation considers shared values relating to children’s health, the presence of a medical school, strength in biomedical engineering, and the quality and scope of ongoing research. The foundation also considers institutional commitment to providing technical support, as well as to translational approaches that promote rapid clinical application of research results to the patient.

Other institutions that received Hartwell Awards include Boston University, Cornell University, Johns Hopkins University, The University of Michigan, St. Jude Children’s Research Hospital, University of California-Davis, the University of Virginia, and The University of Wisconsin.