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ASGCT 2025: Physicians and scientists raced to make a bespoke gene editing therapy for an infant who urgently needed one in months - a first hand account, and what this means for drug development

  • blonca9
  • 15 hours ago
  • 1 min read

As published in NEJM today, UPenn's Kiran Musunuru describes how the team used base editing in a matter of months to treat an infant with CPS1 deficiently, and his thoughts on what the learnings of this case are for drug development.







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