1. The Future Is Now: Gene Therapy for Inherited Retinal Diseases

3. Activity Components

4. An Overview of Inherited Retinal Diseases: Etiology, Symptoms, Prognosis

5. Stargardt Disease: Clinical Features

6. Best Disease: Clinical Features

7. Retinitis Pigmentosa: Clinical Features

8. Retinitis Pigmentosa: Clinical Management

9. Leber Congenital Amaurosis (LCA): Clinical Features

10. Voretigene Neparvovec for the Treatment of Leber Congenital Amaurosis (LCA)

11. Concluding Remarks

12. Gene Therapy: Genetic Components and General Considerations

13. Inherited Retinal Diseases (IRDs): An Overview

14. LCA and EORSD Caused by Mutation in RPE65 Gene: Clinical Phenotype

15. Genetic Testing/Confirmation Is Mandatory

16. Genetic Heterogeneity of IRDs

17. What to Do if a Variant Is Suspected in RPE65

18. Pathogenesis of RPE65 Mutations

19. Therapeutic Approaches for IRDs

20. RPE65 Protein Production: Bringing cDNA to Target Using a Vector

21. RPE65 Gene Delivery

22. Gene Therapy: Accessibility to the Patient

23. Concluding Remarks

24. Gene Therapy: Clinical Data and Considerations

25. Phase 3 Trial Voretigene Neparvovec: Study Design

26. Multi-Luminance Mobility Test (MLMT) Example Layout

27. Mean Bilateral MLMT Lux Score and White Light FST for mITT Population

28. Does the Treatment Effect Persist
Does the Treatment Effect Persist? Mean Bilateral MLMT and FST Over 3 Years

29. Four-Year Update on the Phase 3 Voretigene Neparvovec Study

30. Phase 3 Voretigene Neparvovec Study: Safety Results

31. The First FDA-Approved Gene Therapy for an Inherited Disease: Practical Considerations

32. Voretigene Neparvovec: A Medical Breakthrough

33. Pathogenesis of RPE65 Mutations

34. RPE65 Protein Production: Bringing cDNA to Target Using a Vector

35. RPE65 Gene Delivery Procedure

36. Patient Considerations

37. Insurance Approval Is Based on Genetic Testing Results

38. Voretigene Neparvovec: Process of Treatment

39. Concluding Remarks

40. Concluding Remarks: The Future of Gene Therapy in IRDs

41. Voretigene Neparvovec: Patients Treated in French Center of Excellence

42. Abbreviations