FDA approves gene therapy for severe leukocyte adhesion deficiency-I, a rare immune disorder

Summary: Imagine your body is a football team, but your star defenders are stuck on the sidelines without their cleats. That is exactly what happens to children with a very rare disease called severe LAD-I. Because of a tiny mistake in their DNA (the ITGB2 gene), their white blood cells are missing two important proteins called CD11 and CD18. Without them, the cells cannot travel to fight off germs, leaving the kids wide open to dangerous, life-threatening infections.

For over 30 years, Dr. Donald Kohn at UCLA has been working on a way to get those defenders back in the game. Now, the FDA has officially approved a groundbreaking gene therapy called Kresladi. Doctors take the patient's own blood stem cells, insert a healthy copy of the gene, and give them back. In the clinical trial, it worked perfectly. All the children survived without needing a risky bone marrow transplant, and their severe infections dropped dramatically. It is a massive victory for science and gives these kids a real shot at a healthy life.

For more details, see rssapp-uclahealth-org-news at uclahealth.org/news/release/fda-approves-gene-therapy-severe-leukocyte-adhesion (opens in new tab)