An overview of gene therapy by experts from Johns Hopkins Medicine and Kennedy Krieger Institute. The panel will cover core concepts, current uses (including Duchenne Muscular Dystrophy, Hemophilia B, other genetic disorders, and N-of-1 trials), and the clinical and operational setup needed to deliver these treatments safely. Speakers will outline key barriers (cost, risk, access, and follow-up demands), along with the multidisciplinary roles involved in patient care. The session concludes with a brief look at the gene therapy pipeline and what to expect in the years to come. #rarediseaseday #johnshopkins