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Gene Therapy Solutions for Sickle Cell Disease - Lecture 15

The Royal College of Pathologists via YouTube

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This lecture surveys gene therapy strategies for sickle cell disease, explaining viral-vector and CRISPR approaches alongside their risks, clinical results, safety, and durability.

Syllabus

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

Taught by

The Royal College of Pathologists

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