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Philly Doctors Named TIME 100 for Gene Editing Breakthrough
19 Apr
Summary
- Two Philadelphia doctors pioneered life-saving CRISPR gene editing for a baby.
- The therapy treated a rare urea cycle disorder, preventing brain damage.
- Their work opens doors for personalized genetic medicine for children.
Philadelphia doctors Dr. Kiran Musunuru and Dr. Rebecca Ahrens-Nicklas have been named among TIME's 100 Most Influential People of 2026 for their pioneering work in CRISPR gene-editing therapy. This groundbreaking treatment successfully saved the life of KJ Muldoon, an infant born with a rare and life-threatening urea cycle disorder.
The disorder prevents the body from breaking down protein, leading to toxic ammonia buildup that can cause severe neurological damage. Dr. Ahrens-Nicklas of CHOP and Dr. Musunuru of Penn led a joint hospital team that utilized CRISPR technology to identify and edit KJ's defective DNA, replacing it with a repaired sequence.
KJ's condition significantly improved, allowing him to leave the hospital in June 2025 after over 300 days. Nobel Prize-winning biochemist Jennifer Doudna lauded their achievement, stating their work proves on-demand CRISPR therapies can be developed quickly and safely for patients in critical need. Doudna predicted their names would be in medical textbooks for opening the door to personalized genetic medicine.
In February 2026, the Muldoon family and CHOP doctors advocated for increased pediatric research funding on Capitol Hill. This effort aims to expand access to transformational gene therapies for more children suffering from rare diseases.
