In a First, CRISPR Used to Treat Infant With Ultra-Rare Disease

Image: CRISPR-Cas9 is a customizable tool that lets scientists cut and insert small pieces of DNA at precise areas along a DNA strand. […] Image credit: Ernesto del Aguila III, National Human Genome Research Institute, NIH. National Institutes of Health via Flickr. Public domain. Article by Jennifer Henderson. MedPage Today – May 15, 2025. Primary source: New England Journal of Medicine.

Key Takeaways

Two infusions of a customized CRISPR base-editing therapy showed benefit in a baby with an ultra-rare inborn error of metabolism affecting the urea cycle.

Once regulatory approval was obtained, the baby, who was diagnosed with severe carbamoyl-phosphate synthetase 1 (CPS1) deficiency, received infusions of the therapy at approximately 7 and 8 months of age.

In the 7 weeks following the initial infusion, the baby was able to receive an increased amount of dietary protein and a reduced dose of existing medication to half the starting dose, and there were no serious adverse events. […]

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Related articles:

Infant Becomes First Human Ever To Receive Personalized CRISPR Gene Therapy Treatment – IFLScience

First-Ever Custom CRISPR Therapy Saves Infant With Deadly Genetic Disorder – Gizmodo

World First: US Baby Treated With Personalized CRISPR Gene-Editing – ScienceAlert