Optimizing human α‑galactosidase for treatment of Fabry disease
Published in Scientific Reports
Fabry disease is caused by a deficiency of α-galactosidase A (GLA) leading to the lysosomal accumulation of globotriaosylceramide (Gb3) and other glycosphingolipids. Fabry patients experience significant damage to the heart, kidney, and blood vessels that can be fatal. Codexis scientists applied directed evolution to generate more stable and efficacious GLA variants as potential next generation treatments for Fabry disease. In this paper published in Nature, findings from this research highlight the promise of using directed evolution to generate enzyme variants for more effective treatment of lysosomal storage diseases.