Department of Pharmacology, Toxicology & Neuroscience, and Gene Therapy Program, Louisiana State University Health Sciences Center, Shreveport, LA 71130, USA.
Neurotox Res. 2009 Oct;16(3):329-42. doi: 10.1007/s12640-009-9080-7. Epub 2009 Aug 11.
A growing variety of technical approaches allow control over the expression of selected genes in living organisms. The ability to deliver functional exogenous genes involved in neurodegenerative diseases has opened pathological processes to experimental analysis and targeted therapeutic development in rodent and primate preclinical models. Biological adaptability, economic animal use, and reduced model development costs complement improved control over spatial and temporal gene expression compared with conventional transgenic models. A review of viral vector studies, typically adeno-associated virus or lentivirus, for expression of three proteins that are central to major neurodegenerative diseases, will illustrate how this approach has powered new advances and opportunities in CNS disease research.
越来越多的技术方法可以控制生物中特定基因的表达。将参与神经退行性疾病的功能性外源基因递送到啮齿动物和灵长类动物临床前模型中，使研究人员能够对病理过程进行实验分析和靶向治疗开发。与传统的转基因模型相比，病毒载体（通常是腺相关病毒或慢病毒）研究具有更好的生物适应性、更经济的动物利用和更低的模型开发成本，可更精准地控制基因表达的时空特性。本文综述了三种主要神经退行性疾病的关键蛋白的病毒载体表达研究，展示了这一方法如何为 CNS 疾病研究带来新的进展和机遇。
