The term “Gene” (Gene) is the Chinese phonetic name for the basic factor, the essential element. A gene is a carrier of genetic information and the smallest functional unit of genetic material and refers to a specific sequence of nucleotides in a DNA molecule. The Human Genome Project, launched in 1990 and successfully completed in 2003, has unlocked the genetic code and deciphered the human genome. The human genome contains about 30,000 genes, and the analysis of the structure and function of these genes is the real beginning of human understanding of ourselves and life. Gene therapy is simply the use of genes to cure diseases, for example, if a gene in the body is mutated and causes a disease, it is replaced or supplemented with a new correct gene. In theory, such a strategy could not only treat but also cure countless human genetic diseases, but in practice, it is very difficult to develop safe and effective gene therapy. After optic nerve injury, the microenvironment is altered, and some components are altered to protect the optic nerve, such as CNTF, BDNF, and NT-4/5, which are cytokines that enhance the survival of retinal ganglion cells after optic nerve injury. If these cytokines are injected into the eye in the usual way, the effect will be very short-lived, so scientists are trying to use gene therapy by loading these cytokines onto viral or other non-viral vectors and introducing them into the eye, hoping that they can play a long-lasting and stable protective role. Conversely, genes that inhibit nerve growth could be reduced by inhibiting gene expression, thereby indirectly promoting repair of nerve damage, which could benefit not only optic nerve damage, but also patients with various forms of retinal degeneration. These ideas have been validated in cellular and animal studies, but there are still some obstacles to their practical application, among which the choice of gene vectors remains a central issue: the effectiveness and specificity of transfection with non-viral vectors is not ideal, and the vitreous humor also affects the diffusion of these polymers into cells; viral vectors have transfer specificity problems, and the transfection rate of retinal ganglion cells varies greatly In addition, the immune response, toxic effects, and the unavoidable damage caused by vitreous cavity injection are all issues that need to be addressed. Therefore, the clinical application of gene therapy needs to be further investigated in depth.