Ancient Gene Holds Promise for Gout Treatment
BOSTON, MA – Scientists have resurrected a gene lost in human evolution millions of years ago, offering a potential breakthrough in the treatment of gout.Researchers at Harvard University have successfully restored uricase production in human liver cells using CRISPR gene-editing technology, paving the way for a gene therapy that could provide a long-lasting and safer alternative to current gout medications.
Gout,a painful form of inflammatory arthritis,affects an estimated 9.2 million U.S.adults,according to the Centers for Disease Control and Prevention. The condition arises from a buildup of uric acid crystals in the joints. Current treatments, like Krystexxa, involve injections of uricase proteins derived from pig and baboon genes, frequently enough triggering notable immune responses and carrying the risk of anaphylactic shock. This new approach aims to circumvent those issues by reintroducing a version of the uricase gene humans possessed before losing it during evolution.
“Ideally we can use ancient proteins or ancient enzymes to develop therapeutics to help modern society,” said researcher Eric Gaucher, outlining the broader potential of adapting ancient genes for modern medical applications.The team’s work, detailed in recent research, focuses on utilizing nanoparticle delivery systems to introduce CRISPR components directly into liver cells, effectively reactivating the ancient gene.The researchers are currently transitioning from liver spheroids to lab mice to further test the efficacy and safety of this gene therapy.While still in the early stages of advancement, this innovative approach represents a significant step toward a potentially transformative treatment for gout and a novel strategy for tackling other diseases by leveraging the power of our evolutionary past.
