🧪 Precision for One, Promise for Many: CRISPR Meets the Clinic--A New Era in Rare Disease Therapy
Dr RR Baliga's "Got Knowledge Doc" Podkast
May 21, 20256m 43s
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Show Notes
🧬✨ A baby with deadly CPS1 deficiency (which results in elevated ammonia levels) gets a custom-made gene edit—delivered by lipid nanoparticles—in just 6 months! 🚀
Ammonia levels down, no hospitalizations, and hope up.
N-of-1? More like Future-of-Many. 💡👶🔬 #GeneTherapy #PrecisionMedicine #RareDisease