FGCU scientists work to improve treatment for genetic diseases

Improving gene therapy to prevent disease for future generations, FGCU researchers are changing the way we treat genetic diseases. We looked at how they’re doing it and if it’s working.

Cancer. Heart disease. Diabetes. Chances are you have or know someone with one of these genetic diseases.

“We have a lot of genetic diseases that are not treatable,” said Arsalan Mirjafari, an FGCU associate professor of chemistry.

But FGCU researchers believe they’re on the verge of changing that. Mirjafari and his undergraduate team of chemists are working to improve gene therapy.

“This project specifically is for gene delivery vectors, which is just stuff that will carry DNA into cells,” said David Siegel, an undergraduate research assistant at FGCU.

MORE: FGCU published project findings

Siegel said the chemical compounds he makes brings the good DNA into cells and pulls the bad DNA out. While others already do that, Siegel adds their methods aren’t always safe.

“They use viruses right now because viruses like COVID take DNA, put it in your cells and replicate it,” Siegel said. “But then you’re stuck with a virus … Sometimes they mutate, and sometimes they’ve caused people harm in the past.”

But FGCU scientists say their compounds can’t mutate. They’re less toxic and more efficient, and they’ve seen results.

“Hopefully, one day those chemicals will change lives,” Mirjafari said.

FGCU recently earned a $250,000 grant from the National Science Foundation to continue its work on gene therapy as well as several other projects.

MORE:

• FDA – What Is Gene Therapy? How Does It Work?
• National Science Foundation – FGCU research project