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Crispr treatmentWired
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Health
70% Informative
KJ Muldoon has a rare disorder known as CPS1 deficiency, which causes ammonia to build up in the blood.
About half of babies born with it will die early in life.
A team at the Children’s Hospital of Philadelphia and Penn Medicine used Crispr to create a personalized medicine for KJ .
KJ is now able to eat certain foods and sit upright by himself.
KJ , a 27-year-old with Duchenne muscular dystrophy, passed away in 2022 shortly after receiving the first known custom Crispr treatment.
Researchers used a version of Crispr called base editing that can change one “letter” in a DNA sequence to another.
They packaged the Crispr components in tiny bubbles called lipid nanoparticles, which were then delivered via an IV infusion.
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