The pioneering base-editing method of Alexis Komor, PhD, was recently used to create a custom therapy for an infant with carbamoyl phosphate synthetase 1, a deadly rare disease. Her colleague, Gene Yeo, PhD, favors another new approach: RNA-targeting precision therapeutics, which he used to target ALS & aid a child with developmental epileptic encephalopathy. Both employ AI to discover the roots of polygenic diseases—from the ultra-common like diabetes & heart disease to the ultra-rare—& decode the Dark Genome. More on the promise the technology holds for the future of personalized treatment.

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