1. Gene Therapy: Past, Present, and Future

3. This program will include a discussion of off-label treatment and investigational agents not approved by the FDA for use in the United States.

4. Overview of Hemophilia Treatment

5. History of Gene Therapy in Hemophilia

6. AAV Vector

7. First Wave of AAV Clinical Trials

8. First Wave of AAV Clinical Trials (cont)

9. Advances Following the First Wave

10. AAV8 Vector in Hemophilia B

11. Host-Immune Response

12. Innovations in Gene Therapy in Other Diseases

13. Current Clinical Trials in Hemophilia B

14. Current Clinical Trial in Hemophilia A

15. The Future

16. Gene Therapy and the Treatment Paradigm

17. Gene Therapy and Children

18. Applicability of the AAV Vector

19. Ongoing Concerns

20. Identifying Endpoints for Clinical Trials

21. Other Technologies

22. Concluding Remarks

23. Abbreviations