Gene Therapy Solutions for Sickle Cell Disease - Lecture 15

Gene Therapy Solutions for Sickle Cell Disease - Lecture 15

Explore the cutting-edge field of gene therapy for sickle cell disease in this comprehensive lecture. Delve into various gene therapy solutions, underlying technologies, and notable pre-clinical and clinical achievements. Gain insights into the potential risks and benefits associated with gene therapy for hemoglobin disorders. Learn about antiviral vectors, CRISPR systems, DNA break mechanisms, and genotoxicity concerns. Examine clinical trials, including BB305, BC11A, and G206, and their results, safety profiles, and durability. Analyze hemolysis markers and understand the current challenges and future prospects of gene therapy in treating sickle cell disease.