One-of-One CRISPR: How Scientists Saved a Baby with Personalized Gene Editing

Published on Spencer Knight via LinkedIn

A landmark case in gene-editing has just unfolded: baby KJ, only nine months old and suffering from a unique CPS1 deficiency, received a bespoke CRISPR base-editing therapy developed entirely from scratch in under a year. Designed by teams at the University of Pennsylvania and Children’s Hospital of Philadelphia, this one-of-a-kind treatment corrected a single-letter mutation in KJ’s DNA. The result: KJ’s ammonia levels have stabilized, and the therapy continues to work, giving hope that “n‑of‑1” personalized gene therapies can become a real option

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👉 Spencer Knight: CRISPR saves baby’s life with custom therapy

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