A baby named KJ, born with a life-threatening genetic disorder, is the first person to receive a personalized CRISPR gene-editing treatment. ⁣ Without the CPS1 enzyme, ammonia builds up in the blood, causing brain damage—over half of affected infants don’t survive. Researchers Rebecca Ahrens-Nicklas (Children’s Hospital of Philadelphia) and Kiran Musunuru (University of Pennsylvania) rapidly developed a base-editing therapy targeting one of KJ’s gene copies. ⁣ He received his first dose in February 2025, followed by higher doses in March and April. Since then, he’s shown early signs of improvement, eating more protein with fewer medications, though it’s still too early to assess long-term results. ⁣ KJ’s case marks a major step toward personalized medicine for rare genetic conditions, using gene editing tailored to a unique mutation. ⁣ #CRISPR #Genetics #Medicine #Innovation #GeneEditing #Healthcare #Biotech #Science #Breakthrough #Treatment