Key Takeaways:
• Baby KJ Muldoon received a custom base-editing cure for CPS1 deficiency at CHOP in 2025 — a single-letter DNA fix delivered via IV, avoiding a liver transplant. He took his first steps before Christmas.
• Base editing (a precise CRISPR variant that doesn't cut DNA) is ideal for point mutations. CRISPR-Cas9 works best for gene knockout. Prime editing (the most versatile) enters trials in 2026.
• The FDA approved KJ's individual therapy in one week. The Innovative Genomics Institute is building a "cookbook" to make on-demand custom therapies repeatable and faster.
• Gene editing is most accessible today for confirmed point mutations in reachable organs like the liver. Nome evaluates whether your child's mutation qualifies — submit your diagnosis for a free analysis.
