JACKSONVILLE, Fla. – Jacksonville may be making history in the world of biotechnology.
A local research group is preparing to launch a first-of-its-kind gene editing study aimed at helping people with severe cholesterol conditions, and it could permanently change how the disease is treated.
Gene editing refers to a group of technologies that allow scientists to precisely add, remove, or alter DNA within an organism’s genome. Researchers are now applying that technology to tackle serious cholesterol problems through the liver.
Dr. Michael Koren, with Encore Research Group of Jacksonville Center for Clinical Research, is set to lead the study.
“We could be the first place in the United States, in all of the U.S., to do gene editing studies for cholesterol issues,” Koren said.
While some cholesterol conditions develop over time, others are inherited.
One such condition is familial hypercholesterolemia, a genetic disorder that causes dangerously high cholesterol levels from birth.
“There is a genetic type of cholesterol problem called familial hypercholesterolemia that affects between 1 in 300 and 1 in 500 people in the U.S.,” Koren said.
People diagnosed with the disorder often face serious health consequences early in life, including the potential need for surgery or a heart attack before age 65.
While medications can help manage cholesterol levels, this study marks the first time a therapy has aimed to reverse the underlying problem rather than simply treat its symptoms.
The new therapy would permanently lower cholesterol levels, potentially reducing the risk of heart attacks and other dangerous health complications.
Participants in the study will be monitored for 15 years.