Research
Breakthroughs
CMB
Faculty
The National Institutes of Health (NIH) recently awarded JABSOM's Jesse Owens a highly competitive R01 grant for his lab’s groundbreaking work in gene editing technology.
“This is my dream grant,” said Owens, associate professor at JABSOM’s Dept of Cell and Molecular Biology. “It’s the project I’ve always wanted to do. It funds exactly what our lab is passionate about, which is developing safer, more precise tools for gene therapy that can be used across many different diseases.”
The four-year, $2 million grant supports the continued development of a new generation of transposases, the specialized enzymes that allow scientists to insert genes into precise locations within the genome. Unlike other gene-editing approaches, such as CRISPR, which make cuts to DNA, Owens’ method focuses on delivering genes with extraordinary accuracy.
That precision has been years in the making. One of Owens’ graduate students, Chris Tran, painstakingly created and tested more than 200 enzyme mutants to identify one with extremely low off-target activity. The next step, Owens explained, is to optimize the system to increase its “on-target” efficiency, the rate at which genes land exactly where they’re intended.
“Our goal now is to find that perfect balance. We’ve minimized the off-target effects, now we’re working on boosting the on-target performance so that the system is both incredibly safe and incredibly effective,” he said.
Owens’ lab has already made remarkable progress. When he began, his team was achieving gene delivery efficiencies of less than one percent. Through years of fine-tuning, their latest system has reached ~100 percent efficiency. This leap that would have seemed impossible just a few years ago.
“What we didn’t realize early on was just how fine-tuned this system needed to be,” Owens said. “If you move the target by just two base pairs, the efficiency can drop dramatically. We had to test hundreds of iterations to find the right combination. It was a lot of hard work but it’s what made our tool so precise.”
The R01 grant supports exactly this kind of tool-building research which allows the development of new methods and technologies that other scientists can use to advance their own disease-specific studies. Owens describes his lab as “disease agnostic,” and builds platforms that can be applied to a wide range of conditions, from hemophilia to cystic fibrosis to cancer.
“It’s a special type of R01,” he explained. “It’s not tied to one disease area, which is perfect for us. We can focus on making the best tool possible, and then share it with researchers who specialize in different diseases. Science moves forward when everyone is playing to their strengths.”
Ultimately, Owens hopes the technology will accelerate progress in CAR T immunotherapy, an innovative approach that reprograms a patient’s own immune cells to target and destroy cancer. His team plans to test its system in human T-cells to ensure safety and effectiveness before collaborating with clinical researchers.
“The really exciting thing is that this could one day help treat actual patients. Imagine something that started in your PhD eventually becoming part of a therapy that fights cancer,” Owens said. “That’s what drives us.”
Beyond the science, the grant also ensures steady support for the next generation of biomedical researchers. Two JABSOM graduate students will be fully funded to continue working on the project, giving them hands-on experience at the leading edge of gene therapy innovation.
“This grant keeps our students doing the projects they’re truly passionate about,” Owens said. “They get to see how their hard work contributes to something bigger, something that could ultimately improve human health.”
For JABSOM, the award marks another milestone in advancing Hawaiʻi’s leadership in molecular medicine. Owens’ lab has already seen its earlier technologies adopted by academic groups and biotech companies worldwide, proof that discoveries made in Hawaiʻi are making an impact far beyond the islands.
“JABSOM is proud to be part of this moment,” said Dean Sam Shomaker. “Dr. Owens and his team are not only advancing the science of gene editing, they’re inspiring the next generation of scientists who will continue our legacy of innovation and discovery.”