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Custom gene editing shows promise for ultrarare diseases
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2026 6 months ago
Last year, a baby boy with a life-threatening metabolic condition became the world’s first patient to receive a personalized gene-editing treatment. The feat could pave the way for gene editors tailored to people with unique or ultrarare mutations.
CREDITS: (FOOTAGE) CHILDREN’S HOSPITAL OF PHILADELPHIA; (ILLUSTRATION) A. MASTIN/SCIENCE; (VIDEO PRODUCTION) M. CANTWELL/SCIENCE
#CRISPR #Science #ScienceShorts
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