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

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

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Main Authors: Naomi Chadderton, Arpad Palfi, Daniel M. Maloney, Matthew Carrigan, Laura K. Finnegan, Killian S. Hanlon, Ciara Shortall, Mary O’Reilly, Peter Humphries, Lorraine Cassidy, Paul F. Kenna, Sophia Millington-Ward, G. Jane Farrar
Format: Article
Language:English
Published: MDPI AG 2023-01-01
Series:Pharmaceutics
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Online Access:https://www.mdpi.com/1999-4923/15/2/322