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4. Single Systemic Administration of a Gene Therapy Leading to Disease Treatment in Metachromatic LeukodystrophyArsaKnock-Out Mice

5. Gene therapy for metachromatic leukodystrophy: Lead candidate optimization

9. Papulosquamous Diseases

10. Deposition Disorders

12. Vascular Anomalies

19. Fibrous Proliferations

23. Cutaneous Metastases

24. Fungal Diseases

26. Interface Dermatoses

27. Spongiotic Dermatitis

28. Nutritional Disorders

33. Granulomatous Eruptions

35. Panniculitis

38. Neural Proliferations

44. HMI-202: A gene therapy development candidate for metachromatic leukodystrophy (MLD)

48. Sustained Correction of a Murine Model of Phenylketonuria following a Single Intravenous Administration of AAVHSC15-PAH

50. HMI-202: Investigational gene therapy for treatment of metachromatic leukodystrophy (MLD)

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