Brain-derived neurotrophic factor (BDNF) exerts its survival-promoting effects on photoreceptors and retinal ganglion cells, however, delivery systems with little-to-no side effect are needed to sustain its controlled release and long-term efficacy. Our previous studies demonstrated that adipose-derived stem cells (ADSCs) are ideal delivery systems for gene therapy; moreover, ADSCs present unique properties like migration to damaged tissue sites, immunomodulation and anti-inflammation. Herein, we propose to employ ADSCs as the BDNF gene delivery vehicle. Different Analyses like flow cytometry, differentiation and cell proliferation assays etc demonstrated that BDNF were successfully transduced into ADSCs and the stemness of ADSCs was maintained even with the transduction. Real Time PCR and Western blot were used to measure mRNA and protein expressions of the BDNF-transduced ADSCs. The results demonstrated that the BDNF expression level of the lentiviral-BDNF transduced ADSCs is significantly increased and, moreover, enhanced the expression of other neurotrophic and downstream signaling factors. The data suggest that ADSCs are a reliable delivery vehicle for BDNF and could be used for the treatment of various diseases.

中文翻译：

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携带脑源性神经营养因子基因的慢病毒载体转导的脂肪源间充质干细胞的体外研究。

脑源性神经营养因子（BDNF）对光感受器和视网膜神经节细胞发挥其生存促进作用，但是，需要几乎没有副作用的给药系统来维持其控释和长期疗效。我们以前的研究表明，脂肪干细胞（ADSCs）是基因治疗的理想递送系统。此外，ADSC具有独特的特性，例如迁移到受损的组织部位，免疫调节和抗发炎。在本文中，我们建议采用ADSC作为BDNF基因递送载体。流式细胞术，分化和细胞增殖测定等不同分析表明，BDNF已成功地转导至ADSC中，并且即使进行转导也保持了ADSC的干性。实时荧光定量PCR和蛋白质印迹用于测量BDNF转导的ADSC的mRNA和蛋白表达。结果表明，慢病毒-BDNF转导的ADSC的BDNF表达水平显着增加，此外，增强了其他神经营养和下游信号传导因子的表达。数据表明，ADSC是BDNF的可靠传递载体，可用于治疗各种疾病。