Longevity & AgingAI-Designed Gene Therapy Extends Lifespan 83% in Progeria Mouse Models
Researchers used artificial intelligence to design a new genetic therapy called LM2556 for progeria — a rare disease causing extreme premature aging and early death, mainly from heart disease. The therapy works by targeting a specific region of the gene responsible for producing toxic proteins that damage cells and accelerate aging. Tested in patient-derived heart cells, 3D cardiac organoids, and mice, LM2556 successfully reduced the harmful proteins, reversed cellular aging signs, and improved heart structure and function. Remarkably, treated mice lived 83% longer on average. The treatment showed no signs of liver or kidney toxicity, suggesting a favorable safety profile. This represents a significant advance in both progeria treatment and AI-guided drug design.
