Researchers at Stanford Medicine found an unexpected benefit in a mouse study of Friedreich’s ataxia, a genetic disease that affects the body’s energy-producing mitochondria. After a bone-marrow transplant, healthy immune cells moved into tissues around the body.
The transplanted cells then handed some of their healthy mitochondria to nearby cells in the heart and brain. Mitochondria act like tiny power stations inside cells, so this transfer helped restore some energy-related activity.
Treated mice showed better growth, coordination, muscle strength, heart function, and survival than untreated mice in the study. The improvement was partial, and the work has not yet shown that the approach is safe or effective in people.
The researchers say the finding could point toward treatments for several mitochondrial disorders. It may also help scientists think about ways to deliver gene-based treatments throughout the body.
