Medical Xpress·3 min read·hard
Liver-directed gene therapy shows preclinical efficacy for severe inherited metabolic disorder
T
Telethon Foundation
✦AI Summary
Researchers have published preclinical data showing that a liver-directed gene therapy can effectively treat methylmalonic acidemia (MMA) in mice. The study demonstrates sustained improvements in disease markers, offering hope for a future treatment for this rare metabolic disorder.
edited by Gaby Clark , reviewed by Andrew Zinin
sciencehealth
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