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HHMI
hhmi.org
did:plc:7wfnny4hjpeow4qnevixtnou
A baby named KJ, born with a rare genetic disorder called CPS1 deficiency, is thriving thanks to the world’s first personalized gene-editing treatment. 🧬✨
Doctors at @childrensphila.bsky.social used base editing—a tool invented by HHMI Investigator David Liu and his lab at Harvard.
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2025-05-15T19:17:45.049Z