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Introduction
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Classroom Contents
Gene Therapy Solutions for Sickle Cell Disease - Lecture 15
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- 1 Introduction
- 2 Outline
- 3 Transplant
- 4 Gene therapy
- 5 Risks
- 6 Strategies
- 7 Antiviral vectors
- 8 CRISPR system
- 9 DNA break
- 10 HD vs NHEJ
- 11 Offtarget genotoxicity
- 12 Ontarget genotoxicity
- 13 Base transversion
- 14 Summary
- 15 BB305
- 16 Clinical trials
- 17 Results
- 18 Crisis
- 19 Safety
- 20 Durability
- 21 Conclusion
- 22 BC11A
- 23 G206
- 24 BC118
- 25 Excessile
- 26 Hemolysis markers
