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

- Link: https://www.widthness.com/in-a-first-crispr-used-to-treat-infant-with-ultra-rare-disease/
- Published: 2025-05-17T09:15:00-05:00
- Author: Curated by John Wysocki

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. […] Click here to view original web page at www.medpagetoday.com 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