Gout is one of the oldest known human diseases. It causes painful swelling in the joints when sharp crystals form due to high levels of uric acid in the body.
Now, scientists at Georgia State University may have found a very old way to treat it—by restoring a gene that humans lost millions of years ago.
The research team used CRISPR, a gene-editing tool, to bring back a gene called uricase. Most animals still have this gene.
Uricase helps break down uric acid, a waste product in the blood. Without enough uricase, uric acid builds up, forming crystals in joints and kidneys. This can lead to gout, kidney problems, and other health issues.
Humans and other apes lost the uricase gene around 20 to 29 million years ago.
Some scientists believe this may have helped early primates store fat from fruit sugars, which gave them more energy during times when food was hard to find. But today, this ancient adaptation is causing problems. High uric acid levels are linked to many modern health issues.
Biology professor Eric Gaucher and his team wanted to see what would happen if they reactivated this missing gene. Using CRISPR, they inserted a reconstructed version of the uricase gene into human liver cells.
To their surprise, the cells started breaking down uric acid again. Even more, the liver cells stopped turning fructose—a type of sugar—into fat.
To make sure the results were accurate, the team tested the gene in more advanced lab models called 3D liver spheroids. These small, lab-grown structures act more like real organs. Again, the results were promising.
The uric acid levels dropped, and the enzyme moved into the correct part of the cell, called the peroxisome, where uricase usually works.
Professor Gaucher explained that reactivating uricase helped lower uric acid levels and stopped the cells from creating triglycerides—fats that can build up in the liver. This means the therapy could help not only with gout but also with fatty liver disease.
The dangers of high uric acid go far beyond joint pain. A condition called hyperuricemia, or high uric acid, is linked to serious health problems like high blood pressure and heart disease.
Some studies show that up to half of people with high blood pressure also have high uric acid levels. For those newly diagnosed with high blood pressure, that number may be as high as 90%.
Gaucher believes that lowering uric acid could help prevent many of these health problems at once. Current treatments for gout don’t work for everyone and can have side effects. A gene-editing method that restores uricase in the liver might offer a safer and more effective solution.
The next step is to test the treatment in animals. If that goes well, human trials could follow. Possible ways to deliver the treatment include injections, returning edited liver cells to the patient, or using the same kind of nanoparticles used in some COVID-19 vaccines.
While the results are exciting, there are still challenges. Safety is a major concern when editing genes, and society will need to decide who should have access to these powerful treatments. But if proven safe, this approach could lead to better, longer-lasting relief for millions of people with gout and related conditions.
If you care about pain, please read studies about how to manage gout with a low-purine diet, and a guide to eating right for arthritis.
For more health information, please see recent studies about the link between processed foods and chronic diseases, and avoid these 8 foods to ease arthritis pain.
The study is published in Scientific Reports.
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