AIM: To investigate the effect of gene modification on the differentiation of bone marrow mesenchymal stem cells into neuron in treatment of optic nerve injury.

METHODS: Lentivirus carrying the rat ciliary neurotrophic factor(CNTF)coding sequence was transfected into bone marrow mesenchymal stem cells isolated from rat femur. Rats with optic nerve injury constructed by clamp optic nerve method were randomly divided into control group and study group. On the 4, 7, 10 and 13d after successful modeling, the transfused bone marrow were injected into the vitreous cavity of the study group. The control group was injected with the same amount of normal saline. On the 14^th day after successful modeling, the light reflexes of the two groups of rats, the number of retinal ganglion cells and the expression of glial fibrillary acidic protein(GFAP)and Caspase-3 protein were observed.

RESULTS: The recovery rate of the study group was significantly higher than that of the control group(83% vs 25%, P<0.05). The results showed that the retinal cells in the study group were relatively neat. A small amount of vacuoles were observed; the retinal cells in the control group were not well-structured, and obvious vacuoles were observed, and the number of cells was decreased. The results of Western blot showed that the expression level of GFAP and the expression level of Caspase-3 were higher than that of the rats in the study group.

CONCLUSION: Genetic modification induces bone marrow mesenchymal stem cells can effectively treat optic nerve injury in rats.