For nearly two decades, researcher Jesse Owens and his team at the University of Hawaiʻi at Mānoa John A. Burns School of Medicine (JABSOM) have been pursuing a bold vision: harnessing the natural ability of DNA to move itself to treat genetic disease. Supported by a $2 million grant , their latest breakthrough, published in Nucleic Acids Research , marks a major step forward in precision gene therapy.
In nature, transposons, sometimes called "selfish DNA," can jump around the genome, a phenomenon first discovered in corn by Nobel laureate Barbara McClintock. Owens' team repurposed this mechanism to deliver healthy genes into cells, replacing faulty ones.
"What we've done in our lab is take this natural jumping mechanism and use it to deliver healthy genes into the genome, essentially replacing a faulty one with a working copy," Owens said.
Early transposon systems were limited by random insertion, but Owens' lab engineered a way to target "safe harbor" regions of DNA, avoiding cancer-related genes. Their results found an average of 1.2 successful insertions per cell, a more than thousandfold improvement over previous efforts.
"That means nearly every cell we worked with received the new gene. It's a huge jump," Owens said.
With National Institutes of Health (NIH) funding, the team plans to apply the technology to CAR T-cell immunotherapy, potentially improving cancer treatments.
"This research began here in Hawaiʻi, and it's now on the brink of something that could impact lives worldwide," Owens reflected. "It's exciting to see how far we've come and how much farther we can go."
