Abstract
This commentary systematically examines the pivotal role of non-human primate (NHP) models in advancing gene therapy for optic nerve diseases, alongside the ethical and practical challenges they face. It highlights how synergistic innovations in artificial intelligence (AI), organoid technology, CRISPR-based gene editing, and novel delivery systems are reshaping translational paradigms. NHPs, with their anatomical and functional parallels to human visual systems, serve as a gold-standard models for evaluating therapeutic safety, delivery optimization, and long-term efficacy, enabling breakthroughs in treating conditions such as Leber congenital amaurosis (LCA) and non-arteritic anterior ischemic optic neuropathy (NAION). Despite ethical controversies, prolonged experimental timelines, and interspecies variability, AI-driven virtual screening, physiologically relevant organoid models, and non-viral nanocarriers are reducing reliance on NHPs while enhancing therapeutic precision. The review proposes future directions focusing on adeno-associated virus (AAV) vector refinement, multimodal data integration, and interdisciplinary collaboration to accelerate the translation of neuroregenerative therapies for irreversible vision loss.
Keywords
NHPs, Optic neuropathies, Gene therapy, AAV, Retinal organoids