Rosalind Franklin University researchers hear a breakthrough
By Judy Masterson firstname.lastname@example.org February 13, 2013 8:12PM
Rosalind Franklin University of Medicine and Science research associates Francine Jodelka (left) of Palatine and Anthony Hinrich (right) of Chicago with Dr. Dominik Duelli of Lake Bluff with mice that helped with the break through for preventing deafness and blindness in humans. | Thomas Delany Jr.~ Sun-Times Media
Updated: April 15, 2013 2:13AM
NORTH CHICAGO — A team of researchers at Rosalind Franklin University of Medicine and Science has restored hearing in mice that have the same genetic mutation that causes deafness in many humans.
The research, which offers hope that some types of deafness, including that caused by Usher Syndrome, will one day be prevented or even cured through drug therapy, was published last week in the journal Nature Medicine.
A team led by Michelle Hastings, assistant professor in the Chicago Medical School’s Department of Cell Biology and Anatomy, designed synthetic RNA-like molecules that recognize and correct the expression of the mutated gene, allowing normal development of the sensory hair cells in the inner ear responsible for detecting sound.
Hastings, who has been awarded a grant from the National Institutes of Health to pursue her research, called the tiny bonding molecules, which act as a sort of patch, “a very promising approach.”
“It’s a new type of drug platform,” she said. “We can modify the targeted mutations and protect cells from damage.”
Clinical trials for similar types of injectable drugs are now underway.
“What we found most amazing was the simplicity of use,” Hastings said. “We injected the mice one time, within five days after they were born and rescued hearing to near normal levels at low frequencies.”
Hastings said hearing in the lab mice was rescued at a “functional level.” The mice still could not hear at very high frequencies and by six months of age, their hearing had declined.
“We still have a way to go get complete rescue of hearing,” Hastings said. “But this is an important demonstration that hearing in general, that congenital deafness, can be rescued.
Hearing impairment is the most common sensory disorder, with one in 1,000 newborns affected by congenital deafness. The mice in Hastings’ study were engineered with Usher Syndrome, which causes hearing loss, blindness and problems with balance in 3 to 6 percent of all children who are deaf and another 3 to 6 percent of children who are hard-of-hearing.
The Chicago Medical School team worked in concert with investigators at Louisiana State University Health Sciences Center in New Orleans and Isis Pharmaceuticals in Carlsbad, Calif.
“It’s important to note that in mice, hearing develops after birth,” Hastings said. “We’re intervening very early in their development. But humans develop hearing in utero. There’s going to be some hurdles to cross in timing of intervention. We need to study what is the latest point we could rescue hearing.”
Successful treatment of human genetic disease in animal models, Hastings said, “opens the door to developing similar approaches to target and cure other causes of hearing loss.”
A resident of Lake Bluff, Hastings, 43, holds a Ph.D. in biology from Marquette University. She was a postdoctoral fellow at New York-based Cold Spring Harbor Laboratory before joining the CMS faculty in 2007.
Rosalind Franklin University, which celebrated its centennial last year, specializes in interprofessional medical and health care education, offering a doctor of medicine program through the Chicago Medical School, doctor of podiatric medicine program through the Dr. William M. Scholl College of Podiatric Medicine, and a range of degrees, including a new PharmD program, through its College of Pharmacy.