Home > Browse Issues > Vol.35 No.7
Construction and Verification of Gene Therapy Vector for Lipoprotein Lipase Deficiency Disease
Wang Kailong1, Zheng Libin1, Zhang Fan1, Shen Liangcai1, Libby Andrew2, Li Xuli3, Zhang Jin1*
1College of Life science and Biotechnology, Hebei Normal University of Science & Technology, Qinhuangdao 066004, China; 2Division of Endocrinology, University of Colorado, Colorado 80045, USA; 3The Third Hospital of Qinhua
Abstract: Lipoprotein lipase (LPL) is the rate limiting enzyme for triglycerides hydrolysis， which catalyses the hydrolysis of the triacylglycerol component of chylomicrons and very low density lipoproteins， thereby providing fatty acids and monoacylglycerol for tissue utilization. LPL gene mutation or deletion may affect the activity of LPL， and result in lipid metabolism disorder. Although the LPL deficiency disease is rare， no cure method is developed till now. In this study， the gene therapy construct MSCV-hLPL was made， which could infect muscle cell line (C2C12)， kidney cell line (HEK293T) and pre-adipocyte cell line (3T3-L1) with over 80% efficiency. Nevertheless， active LPL could be detected at the surface of all these three kinds of cells. Then， three types of cells were injected into nude mice， LPL activity increased significantly in the muscle tissues under the injection sites of the 3T3-L1 line. Our results show that MSCV-hLPL could correct the LPL–/– genotype and the adipose tissue may be the best tissue for transplantation in the future. This is a ground-breaking test in LPL deficiency treatment field， which lays a good foundation for using iPSC to correct the LPL deficiency.
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