CRISPR Restores Ancient Gene for Gout & Fatty Liver Prevention
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Scientists Revive Ancient Gene to Combat Gout and Uric Acid Buildup
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Researchers at georgia State University have used CRISPR gene-editing to restore a lost enzyme, uricase, offering a potential new approach to treating gout, kidney disease, and related conditions.
The Ancient roots of Gout
Gout is one of the oldest documented human illnesses.It develops when sharp crystals form inside joints,triggering intense swelling and pain,and is considered a type of arthritis. Researchers at Georgia State University believe they may have uncovered a surprisingly ancient way to address it.
A study published in Scientific Reports on , details how scientists employed CRISPR gene-editing tools to reactivate a gene absent from the human genome for millions of years. The reactivation of this gene resulted in lowered uric acid levels, the primary culprit behind gout and a host of other health problems (“CRISPR-mediated reactivation of uricase in human cells reduces uric acid levels” – scientific Reports).
The missing component is uricase, an enzyme that remains functional in most other animals.
Uricase efficiently breaks down uric acid, a waste product naturally present in the blood. When uric acid levels become excessively high, it can crystallize within joints and kidneys, leading to gout, kidney disease, and a spectrum of associated health issues.
Why Did Humans Lose Uricase?
Humans and other apes lost the functional uricase gene approximately 20 to 29 million years ago (“Uric acid and gout” – Seminars in Nephrology). Some researchers propose this loss may have initially provided a survival advantage.
Dr. Richard Johnson of the University of Colorado and others have theorized, as detailed in Seminars in Nephrology, that elevated uric acid levels aided early primates in converting fruit sugars into fat, offering a crucial energy reserve during periods of food scarcity. This adaptation may have been beneficial in environments where food availability fluctuated.
However, this ancient adaptation now contributes to a range of modern metabolic disorders. This is the problem that Eric Gaucher, a biology professor at Georgia State, and his team sought to address.
“Without uricase, humans are left vulnerable,” said Gaucher, a co-author of the study. “We wanted to see what would happen if we reactivated the broken gene.”
Reintroducing Uricase with CRISPR
The research team utilized CRISPR-Cas9 gene-editing technology to reactivate the dormant uricase gene in human cells grown in the laboratory. CRISPR-Cas9 acts like molecular scissors, allowing scientists to precisely target and modify DNA sequences.
The results demonstrated that the reactivated uricase enzyme effectively broke down uric acid, reducing its levels in the treated cells. This suggests that restoring uricase function could be a viable therapeutic strategy for conditions caused by uric acid buildup