
Baby heads home after groundbreaking CRISPR therapy; a new approach to vision restoration therapies; the science behind nostalgia and your urge to dance – Morning Medical Update
Key Takeaways
- Personalized CRISPR therapy for CPS1 deficiency in a nine-month-old marks a milestone in treating rare pediatric diseases.
- NIH study reveals that future vision therapies may need to retrain brain pathways, not just repair retinal cells.
The top news stories in medicine today.
Nine-month-old KJ Muldoon was discharged this week from Children’s Hospital of Philadelphia (CHOP) after spending more than 300 days receiving the world’s first personalized CRISPR gene editing therapy for CPS1 deficiency, a rare and often fatal metabolic disorder. The therapy, developed by scientists at CHOP and Penn Medicine, corrected a specific mutation in KJ’s genetic code and marks a major milestone in customized treatment for rare pediatric diseases. KJ’s recovery—celebrated with a hospital “clap-out” and police escort—signals new hope for children with life-threatening genetic conditions.
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