EPISODE · May 21, 2025 · 6 MIN
🧪 Precision for One, Promise for Many: CRISPR Meets the Clinic--A New Era in Rare Disease Therapy
from Dr RR Baliga's "Got Knowledge Doc" Podkast · host MasterMedFacts LLC
🧬✨ 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
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🧪 Precision for One, Promise for Many: CRISPR Meets the Clinic--A New Era in Rare Disease Therapy
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