Abstract: The retina is highly susceptible to abnormal changes due to laser accidents, central nervous system or retinal degenerative diseases, which can seriously threaten visual function. There is still no well-developed repair mechanism for mammalian retinal damage, and MG cells, the “stem cells” of the retina, cannot enter the cell cycle spontaneously, and MG cell transdifferentiation-related signalling pathways and regulatory factors play a crucial role in reprogramming the MG genome. Gene editing treatment of the retina enables the production of large numbers of neurons from MG cells in the damaged mammalian retina, replenishing damaged neurons and restoring vision. Gene editing therapy uses vectors such as adenovirus and lentivirus to introduce exogenous genes into the body to regenerate damaged retinal neurons. The advent of gene therapy holds the promise of achieving repair in a single treatment, as opposed to traditional drug treatments that require long-term medication. This article reviews the current status and problems in the application of gene therapy for retinal repair, the signalling pathways regulating optic nerve regeneration, and the future trends of gene therapy for retinal repair. In the future, gene editing therapy will bring profound changes to the research of optic nerve regeneration and repair, and bring a new dawn to the treatment of retinal diseases.
Key words: retinal damage repair; optic nerve regeneration; retinal progenitor cells；gene therapy; gene editing technology
（Acta Laser Biology Sinica, 2023, 32(5): 414-422）