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Human Gene Therapy

Description: This course gives an overview of the field of human gene therapy. It covers the fundamentals of using gene delivery for treating human diseases, and details the viral vectors that are used for gene delivery, including their generation and use, the non-viral approaches that can be used to deliver therapeutic genes, and the methods that are used for controlling therapeutic gene expression.

COURSE OUTLINE

INTRODUCTION
1. Course Information
2. Interface Tour
3. Course Overview
4. Definition

GENE DELIVERY
5. Objectives
6. Delivery of Therapeutic Genes
7. Gene Therapy Targets
8. Delivery Modes
9. Steps in Gene Therapy
10. Gene Therapy Viral Vectors
11. Gene Therapy Non-Viral Vectors
12. Progress Check

VIRAL VECTORS
13. Objectives
14. Retroviral Life Cycle
15. Retroviral Genome Structure
16. Recombinant Retrovirus
17. Application of Retroviral Vectors
18. Adenovirus
19. Application of Adenoviral Therapy
20. Adeno-Associated Virus
21. Comparison of Viral Vectors
22. Progress Check

NON-VIRAL VECTORS
23. Objectives
24. Liposomes
25. Polycation Conjugates
26. Progress Check

GENE REGULATION
27. Objectives
28. Therapeutic Gene Regulation
29. Control of Therapeutic Gene Expression
30. Progress Check

CONCLUSION
31. Summary

COURSE OBJECTIVES

Students completing this course should be able to:

Understand the concept of gene delivery for therapeutic purposes
Specify the three disease groups that are targeted using gene therapy
List the two different modes of delivery
Describe the four steps of gene therapy
List the viral and non-viral vectors used for gene therapy
List the three viruses that are predominantly used for gene therapy
Describe how recombinant viruses are generated for therapeutic use
Explain how these viruses are used in a therapeutic setting
List the advantages and disadvantages of the different viral vector systems
Explain how liposomes are formed and how they are used for gene therapy
Describe how liposomes can be made cell-specific
Comprehend the importance of gene regulation in therapy
Give two examples of methods by which expression of the therapeutic gene is controlled

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