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.

Introduction
Outline
Transplant
Gene therapy
Risks
Strategies
Antiviral vectors
CRISPR system
DNA break
HD vs NHEJ
Offtarget genotoxicity
Ontarget genotoxicity
Base transversion
Summary
BB305
Clinical trials
Results
Crisis
Safety
Durability
Conclusion
BC11A
G206
BC118
Excessile
Hemolysis markers