Efficient gene transfer and expression of biologically active glial cell line-derived neurotrophic factor in rat motoneurons transduced with lentiviral vectors
Efficient gene transfer and expression of biologically active glial cell line-derived neurotrophic factor in rat motoneurons transduced with lentiviral vectors
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DOI：
10.1046/j.1471-4159.2000.0741820.x
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发表时间：
2000-05-01
影响因子：
4.7
通讯作者：
Déglon, N
中科院分区：
文献类型：
--
作者：
Cisterni, C;Henderson, CE;Déglon, N
几项研究表明，人类免疫缺陷病毒1型（HIV 1）为基础的慢病毒载体感染非分裂的大脑和视网膜神经元的能力，高效率和长期表达的转导基因。我们表明，纯化的胚胎运动神经元可以有效地（>95%）转导的文化使用HIV 1为基础的慢病毒载体编码LacZ。在这些条件下观察β-半乳糖苷酶的表达至少9天。此外，用表达胶质细胞系源性神经营养因子的慢病毒载体转导的运动神经元在没有额外营养支持的情况下存活，表明过表达的蛋白质具有生物活性。我们的研究结果表明，慢病毒载体在研究运动神经元中表达的蛋白质的生物学效应和未来运动神经元疾病基因治疗的发展中的潜力。
Several studies have shown the ability of human immunodeficiency virus type 1 (HIV1)-based lentiviral vectors to infect nondividing brain and retinal neurons with high efficiency and long-term expression of the transduced gene. We show that purified embryonic motoneurons can be efficiently (>95%) transduced in culture using an HIV1-based lentiviral vector encoding LacZ. Expression of beta-galactosidase was observed for at least 9 days in these conditions. Furthermore, motoneurons transduced with a lentiviral vector expressing glial cell line-derived neurotrophic factor survived in the absence of additional trophic support, showing that the overexpressed protein was biologically active. Our results demonstrate the potential of lentiviral vectors in studying the biological effects of proteins expressed in motoneurons and in the development of future gene therapy for motoneuron diseases.