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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
Description:
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.

Gene Therapy Solutions for Sickle Cell Disease - Lecture 15

The Royal College of Pathologists
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