Technology developed at the University and published in the Aug. 31 issue of the journal, “Proceedings of the National Academy of Sciences,” has shown that repairing genes in animals is possible.
This is the first time researchers were able to get genes to repair themselves by injecting new, “correct” genes on top of them.
Dr. Clifford Steer, a University professor who worked on the study, said the treatment is like fixing a flat tire with a nail in it. One way to correct the problem is to put on a new tire. But this technology is more like sealing the tire hole.
Researchers injected a synthetic gene into the defective gene, causing the cell to permanently repair itself.
“This is a broad-based technology that can be applied to humans, animals and plants,” said Steer.
Researchers collaborated with the Albert Einstein College of Medicine in New York and were able to fix a genetic disorder in Gunn rats similar to a human genetic disorder.
Because the Albert Einstein College of Medicine has a colony of Gunn rats, Steer asked them about collaborating on the project.
“It was very uncertain because it was a completely new kind of gene therapy,” said Jayanta Roy-Chowdhury, an Albert Einstein College of Medicine professor.
One of Steer’s breakthroughs was a delivery method for the molecule.
“It is not enough to have the drug,” Roy-Chowdhury said. “You have to be able to deliver it.”
The experiments took place at the Albert Einstein College of Medicine while the data was analyzed at both universities.
The genetic disorder in the rats is similar to Crigler-Najjar syndrome in humans. Crigler-Najjar affects fewer than 500 people worldwide, but was selected for study because researchers could study the disorder in rats. The syndrome is a defect in the oxygen-carrying part of the blood that produces jaundice. Infants with the disorder can incur severe brain damage.
Steer said the next step is to get Food and Drug Administration approval for clinical trials.
Roy-Chowdhury said the syndrome tends to appear in homogenous populations such as western Pennsylvanian Amish and Mennonite communities, which will participate in Steer’s clinical trials.
“It causes a great deal of interest in a 21st-century technology being used on a culture that, in many ways, lives back in the 19th century,” Steer said.

Dawn Throener covers the St. Paul campus and welcomes comments at [email protected]. She can also be reached at (612) 627-4070 x3216.