https://jainlabblog.files.wordpress.com/2017/12/pnas_2017.pdf … Isha Jain at UCSF.
This was the coolest thing at BAAM. It may have applications beyond Leigh Syndrome, since we all lose mitochondrial function as we age. Knockout of https://en.wikipedia.org/wiki/NDUFS4 causes neural damage. Low oxygen (like at high altitude) rescues normal function & lifespan.https://twitter.com/lifespanscience/status/1105247621872988160 …
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https://jainlabblog.files.wordpress.com/2017/12/hypoxiatherapy_science_2016.pdf … The hypoxia program allows organisms to survive while consuming less oxygen. Impaired mitochondrial cellular respiration means organisms can get less energy from oxygen. The hypoxic response helps them be "thriftier" with oxygen.
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Jain said that she tried and failed to treat mitochondrial impairment with a small molecule that targeted Hypoxia-Induced Factor, but then she tried hypoxia itself and it worked.
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Illustrates two broader principles: 1.) don't assume that all the best treatments will be drugs; 2.) a simple solution has fewer places to break than a complex one.
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