SMRTR TechMay 18, 2025Scientific American

First Personalized CRISPR Treatment Gives Baby New Lease on Life

SMRTR summary

A baby boy with a rare genetic disease received a groundbreaking CRISPR therapy tailored to his specific mutation. The treatment, developed in just six months, has allowed KJ Muldoon to thrive at 10 months old. While promising, it's too early to call it a cure. This personalized approach could inspire similar treatments for ultra-rare genetic disorders, but scalability and cost remain challenges.

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