Optimisation of AAV-NDI1 Significantly Enhances Its Therapeutic Value for Correcting Retinal Mitochondrial Dysfunction

• Main Authors: Naomi Chadderton (Author), Arpad Palfi (Author), Daniel M. Maloney (Author), Matthew Carrigan (Author), Laura K. Finnegan (Author), Killian S. Hanlon (Author), Ciara Shortall (Author), Mary O'Reilly (Author), Peter Humphries (Author), Lorraine Cassidy (Author), Paul F. Kenna (Author), Sophia Millington-Ward (Author), G. Jane Farrar (Author)
• Format: Book
• Published: MDPI AG, 2023-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

AAV gene therapy for ocular disease has become a reality with the market authorisation of Luxturna^TM for RPE65-linked inherited retinal degenerations and many AAV gene therapies currently undergoing phase III clinical trials. Many ocular disorders have a mitochondrial involvem...