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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

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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.
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Morning Medical Update © kwanchaichaiudom - stock.adobe.com

Morning Medical Update © kwanchaichaiudom - stock.adobe.com

Baby heads home after groundbreaking personalized gene editing therapy

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. NBC10 Philadelphia has more. Also find our initial coverage of the CRISPR therapy here.

NIH maps brain circuits for vision, offering new path for sight restoration

A new National Institutes of Health (NIH) study identifies which brain circuits are essential for visual acuity and how they’re impacted by retinal damage, pointing to the need for vision therapies that go beyond just repairing the eye. Researchers at the National Eye Institute found that X-type neurons in the brain's lateral geniculate nucleus — key for visual sharpness — suffer impaired function after retinal ganglion cell loss, while Y-type neurons tied to motion detection remain largely unaffected. Published in The Journal of Neuroscience, the findings suggest future sight-restoring treatments may need to retrain brain pathways, not just fix retinal cells.

The science behind nostalgia, and your urge to dance

A new study finds that nostalgia may influence our instinct to move rhythmically to music — a behavior with roots dating back over 50,000 years. Researchers say early humans, including Neanderthals and Cro-Magnons, responded to drumming and vocalizations with physical movement. The findings suggest that today’s urge to “groove” is not only emotional but also deeply ancestral, shedding light on how music, memory and movement are intertwined across human history.

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