【摘要】The invention belongs to the biologic medicine filed and relates to a novel gene therapy compound preparation and the use and application method thereof. When adeno-associated virus is used as gene delivery carrier, a certain dose of cytarabine is combined in use, and obviously increases transfer efficiency of normal cells and tumor cells by the adeno-associated virus, advances expression time offoreign gene mediated by adeno-associated virus, obviously increases positive rate and expression level, thereby improving the gene therapy effect employing adeno-associated virus as carrier, particularly the application in gene therapy for diseases, such as ocular fundus disease, genetic disease and tumor. In tumor gene therapy, the invention has functions of inhibiting tumor cell growth, inducing tumor cell apoptosis, and obviously increases the transfer efficiency and expression level of the gene mediated by adeno-associated virus.