Gene Editing Breakthrough: Custom Therapy Treats Rare Infant Disorder

Landmark Gene Therapy for Rare Disease

• 👶 KJ Mulun, an infant born with a rare metabolic disease affecting 1 in 1.3 million, has received a custom-made gene therapy for the first time in history.
• 🧬 The treatment utilized CRISPR technology to identify and correct a single mutated gene out of the 20,000 in KJ's body.
• 🏥 Developed by teams at the Children's Hospital of Philadelphia and Penn Medicine, this therapy offers hope where previously only a liver transplant was considered a long-term solution.

Prognosis Before and After Treatment

• ⚠️ Before the gene therapy, KJ's prognosis was considered