Gene Therapy Advances and Challenges What are the most recent breakthroughs in gene therapy and how might they change the, Landscape of treating genetic disorders and rare diseases in the near future? Recent Breakthroughs in Gene Therapy 1. Sickle Cell Disease & Beta-Thalassemia Casgevy (CRISPR-based therapy): Edits patient’s stem cells to reduce sickle cell crises. […]