Baby KJ Becomes First Patient to Receive Fully Personalized CRISPR Therapy

Published on Spencer Knight via LinkedIn

At just nine months old, KJ was diagnosed with CPS1 deficiency, a rare metabolic disorder causing toxic ammonia buildup with no existing cure. His mutation was completely unique. In less than a year, researchers from Penn and CHOP developed a one-of-a-kind CRISPR therapy tailored specifically to correct the single-letter error in KJ’s DNA.

In February, KJ became the first human to receive a custom-built gene edit. Months later, his ammonia levels are stable and the treatment is working. Spencer Knight calls this a breakthrough moment not just for CRISPR, but for the future of n-of-1 therapies that could redefine rare disease treatment.

Read the full article via New Scientist:

👉 Baby with rare disease given world-first personal CRISPR gene therapy

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