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This chapter details the story of Baby KJ, who was diagnosed with a rare genetic disease and successfully treated with experimental gene editing technology. The treatment involved a complex process, including genome sequencing and the creation of a customized therapy delivered via lipid nanoparticles. The results were remarkable, with KJ showing significant improvement and no longer needing a liver transplant.
- Baby KJ was diagnosed with a rare genetic disease (CPS1 mutation).
- Experimental gene editing therapy was successfully used to treat KJ.
- The therapy involved creating a customized treatment delivered via lipid nanoparticles.
- KJ showed significant improvement and no longer needs a liver transplant.
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