1. Exploring the Use of Adeno-Associated Virus for Gene Therapy

2. Types of Gene Therapy

3. Rationale for Gene Therapy

4. Current Gene Therapy Vectors

5. Adeno-Associated Virus vs Vector

6. Different AAV Serotypes

7. Benefits of AAV

8. Historical View of AAV

9. AAV Gene Therapies

10. Practical Application of AAV Therapy

11. Overview of Early AAV Clinical Trials

12. Ongoing AAV Clinical Trials

13. Use of AAV in Patients With Hemophilia B

14. Use of AAV in Patients With Hemophilia B Study Results

15. Use of AAV in Patients With SMA

16. Examining SMA Genetics

17. Examining SMA Genetics (cont)

18. Disease Course of SMA

19. Challenges of CNS Delivery of Current Treatments

20. Characteristics of AVXS-101

21. AVXS-101 Clinical Trial in SMA Type 1 Results

22. AVXS-101 Clinical Trial in SMA Type 1 Results (cont)

23. Challenges to AAV Gene Therapy

24. Importance of Multidisciplinary Approach

25. Complications of Immune Response

26. Immune Response of Factor IX

27. Strategies to Combat Immune Response

28. Follistatin Gene Therapy Trial Patients With Becker Muscular Dystrophy

29. Current Barriers Under Investigation

30. Key Elements for Successful Gene Therapy

31. Concluding Remarks

32. Abbreviations

33. Abbreviations (cont)