
Retinal neurodegenerative disease is currently the main cause of irreversible blindness, and there is no effective treatment. Recent years, the advances in cell therapy, gene therapy, tissue engineering and other disciplines have brought new directions for the treatment of these diseases. In fact, due to its organ specificity, the eye has also become the pioneer organ for the clinical transformation of the cutting-edge technologies. The first reported human clinical trial of embryonic stem cells, the first human clinical trial of induced pluripotent stem cells, and the first US FDA-approved gene therapy drug were all used in the treatment of retinal neurodegenerative diseases. Unfortunately, the efficiency of clinical translation remains very low.
Therefore, there is an urgent need for a standardized and reproducible related transformation technology system. It mainly includes the construction technology of animal models of retinal neurodegenerative diseases, the evaluation technology of visual function suitable for animals, the in vivo detection technology of retinal anatomy, and the retinal transplantation technology that has been carried out in early clinical trials. This collection will focus on the above technical systems and provide the technical basis and mature path of preclinical research and early clinical research for the clinical translation of various cutting-edge technologies in ophthalmology.