Isha Jain, PhD, and her team developed a drug that mimics the effects of breathing low oxygen, which could be life-saving for people with mitochondrial diseases who rarely survive past childhood. In mice with Leigh Syndrome—the most common childhood mitochondrial disease—the drug extended lifespan three-fold and reversed symptoms, even when given during late stages of disease. The new drug shows the promise of gas-based therapies for mitochondrial diseases and other common brain and cardiovascular conditions for which low oxygen has been shown to be beneficial.

In this video, Jain and research associate Skyler Blume explain the science behind their discovery and the impact it could have for mitochondrial diseases. Read more about this discovery.

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co-first author Megan Ostrowski, left, and senior author Vijay Ramani on the right
Article January 21, 2025

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