Basil Hubbard, a professor at the University of Alberta, developed a way to make proteins in genetic more precise when they alter DNA code. Photo by University of Alberta
EDMONTON, Alberta, Aug. 11 (UPI) -- Working to move genome engineering closer to medical application, researchers have found a way to refine the way proteins edit DNA to prevent altering the wrong gene, according to a new study.
Clinical trials are already underway using agents that edit DNA sequences to correct for disease, and much of that has been research into single-gene diseases. Researchers said a precise way of editing series of genes is essential -- because editing the wrong thing can have catastrophic effects.
"Whereas traditional pharmaceutical drugs have a transient effect, gene editing could possibly provide a permanent cure for a lot of different diseases," said Basil Hubbard, an assistant professor of pharmacology at the University of Alberta, in a press release. "We still have to overcome many hurdles but I think this technology definitely has the potential to be transformative in medicine."
Researchers compare genome engineering to computer programmers correcting code, with the intent being to replace or repair a section that is missing or incorrect. Researchers use chemical agents containing DNA binding proteins attached to tools that alter genes. While they say the agents generally correct the right gene, it doesn't happen 100 percent of the time because the agents are not precise enough yet.
In the new study, published in Nature: Methods, researchers found a way to autonomously evolve the editing proteins, called transcription activator-like effector nucleases, or talens, to make them more specific and targeted over time.
"This technology allows you to systematically say, 'I want to target this DNA sequence, and I don't want to target these others,' and it basically evolves a protein to do just that," Hubbard said. "Using this system, we can produce gene editing tools that are 100 times more specific for their target sequence."
Hubbard said that with clinical trials already underway for some forms of genome editing, it's likely genome editing will begin to be used for treatment of genetic diseases sometime in the next decade. "We still have to overcome many hurdles but I think this technology definitely has the potential to be transformative in medicine," he said.
