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Commentary Open Access
Volume 7 | Issue 1 | DOI: https://doi.org/10.46439/ophthalmology.7.040

Non-human primate models and multimodal technologies synergistically drive translational breakthroughs in gene therapy for optic nerve diseases

  • 1Henan Academy of Innovations in Medical Science, Zhengzhou Aviation Port Economic Zone, No. 1, Bio- Technology Street, Henan, 450008 China
+ Affiliations - Affiliations

Corresponding Author

Jian Wu, karena.wu@foxmail.com

Received Date: May 21, 2024

Accepted Date: June 25, 2025

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

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