The word “gene” (Gene) is translated from its Chinese phonetic name, which means basic factor, basic element. A gene is the carrier of genetic information and the smallest functional unit of genetic material, and refers to a specific nucleotide sequence in the DNA molecule. Human beings have 24 different DNA molecules, which contain about 3 billion pairs of bases, i.e. nucleotides, which contain all the genetic code of human beings and even the mystery of human birth, old age, illness and death. 1990 launched and successfully completed in 2003, the Human Genome Project, has unlocked the genetic code and deciphered the heavenly book of human beings themselves. The human genome contains about 30,000 genes, and analyzing the structure and function of these genes is the real beginning of human beings’ understanding of themselves and life. Gene therapy is simply the use of genes to cure diseases. For example, if a gene in the body has a mutation that causes a disease, a new correct gene is used to replace or supplement it. In theory, such a strategy could not only treat but also cure countless human genetic diseases, but in practice, developing safe and effective gene therapy has been very difficult. When the optic nerve is damaged, the microenvironment is altered, and some components are altered to protect the optic nerve, such as CNTF, BDNF, and NT1/4/5, which are cytokines that enhance the survival of retinal ganglion cells after optic nerve damage. If these cytokines are injected into the eye in the same way as ordinary drugs, the effect will be very short-lived, so scientists are trying to use gene therapy to load these cytokines onto viral or other non-viral vectors and introduce them into the eye, in the hope of exerting a long-lasting and stable protective effect. In turn, some of the genes that inhibit nerve growth, the use of inhibition of gene expression method to make its role weakened, thus indirectly promoting the repair of nerve damage, this therapy not only can make the optic nerve damage to be treated, but also can make a variety of retinal degeneration patients benefit. These ideas have been validated in cellular and animal tests, but there are still some obstacles in practical application, in which the choice of gene vectors is still the core problem: the effect and specificity of transfection with non-viral vectors are not ideal, and the vitreous body also affects the diffusion of these polymers into the cells; and viral vectors have the problem of transfer specificity, and the rate of transfection of retinal ganglion cells varies greatly! In addition, immune response, toxicity, and unavoidable damage caused by vitreous injection are all problems that need to be solved. Therefore, the clinical application of gene therapy requires further in-depth research.