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In little Leo, the face of future personalized medicine

A California biotech has leveraged artificial intelligence and other cutting-edge tools in a perilous rescue mission focused on a 2-year-old Setauket boy born with an unusual genetic disease.

STAT News: Can AI build custom ‘n-of-1’ drugs faster?

Two years ago, Yiwei She was searching desperately for someone to build a drug for her newborn son, Leo. Leo was born with a very small head, a symptom of many severe diseases. After a seizure at two months, doctors sequenced his genome and found a single misspelling in a gene called TNPO2. 

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