Another life-saving treatment from Dr. Donald Kohn's group at UCLA and collaborators. Congratulations to the incredible team of investigators. Another milestone for the miracles of cell and gene therapy.
A new study in @nejm.org reports that an investigational gene therapy restored immune function in 9 children with LAD-I, a rare & life-threatening immune disorder. Those children are now living symptom-free 2 years after treatment in clinical trials co-led by Dr. Donald Kohn at UCLA.
