NEJM: First In Vivo Base Editing Therapy for Infant With Rare Genetic Disorder

Published on New England Journal of Medicine (May 15, 2025)

A nine-month-old patient with fatal CPS1 deficiency has become the first human to receive a patient-specific CRISPR base-editing therapy delivered in vivo. Developed and approved within months, the lipid nanoparticle treatment allowed the child to tolerate more dietary protein and reduce medication dosage with no serious adverse events. The study, led by CHOP and Penn researchers and funded by NIH, represents a turning point in how ultra-rare genetic diseases might be treated in real time.

Read the full article on NEJM:

👉 Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease

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