Dr. Donald Kohn has been developing gene therapies for rare pediatric immune disorders for over 30 years. This week, his role in a clinical trial has culminated in the first-ever U.S. Food and Drug Administration-approved therapy for severe leukocyte adhesion deficiency-I — a genetic condition characterized by recurrent infections and, often, early death.
https://www.uclahealth.org/news/release/fda-approves-gene-therapy-severe-leukocyte-adhesion