2025 Best Baby Savers
Rebecca Ahrens-Nicklas and Kiran Musunuru
These two physicians led the CHOP and Penn Medicine team that, in May, made medical history by successfully using CRISPR gene-editing therapy to treat an infant born with a rare metabolic disease. Baby KJ was only months old when the team started administering the individually tailored therapy for his CPS1, or severe carbamoyl phosphate synthetase 1 deficiency. The landmark gene-correcting approach spares KJ the need for a liver transplant down the road and prevents neurological damage that could prove fatal. Doctors say he’s now growing well and thriving. Read More »