Gene-Editing Breakthrough Cures Infant’s CPS1 Deficiency in World First
Boston, USA: Doctors announced the first successful personalized CRISPR treatment for a baby with CPS1 deficiency—a fatal metabolic disorder affecting 1 in 1.3 million births. In under six months, researchers designed a custom RNA guide, delivered it via lipid nanoparticles, and restored liver enzyme function, allowing ammonia levels to normalize. Regulators fast-tracked compassionate-use approval; the infant is thriving at home.
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Key Entities
- • CRISPR Technology – Gene-editing method using Cas enzymes. Second sentence: Enables targeted DNA changes with unprecedented precision.
- • CPS1 Deficiency – Rare urea-cycle disorder causing toxic ammonia buildup. Second sentence: Untreated infants face organ failure within weeks.
- • FDA Compassionate-Use Program – Allows experimental treatments for life-threatening conditions. Second sentence: Expedites access while collecting safety data.
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Advocates wide public funding and equitable access.
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Focuses on scientific milestone and regulatory path.
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Highlights private-sector innovation and cost concerns.
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