Martin-Rendon E, Azzouz M, Mazarakis N D
Oxford BioMedica, UK.
Curr Opin Mol Ther. 2001 Oct;3(5):476-81.
A number of potential gene therapy applications in the adult nervous system include neurodegenerative disorders, such as Alzheimer's disease, Parkinson's disease and amyotrophic lateral sclerosis. During the last five years, lentiviral vectors have developed into extremely efficient gene transfer vehicles to the nervous system, revealing a wide range of possibilities for the treatment or such disorders. This review describes the most important and recent advances in the development of lentiviral vectors as well as the demonstration of proof-of-principle in animal models of human neurodegenerative diseases.
在成人神经系统中,许多潜在的基因治疗应用包括神经退行性疾病,如阿尔茨海默病、帕金森病和肌萎缩侧索硬化症。在过去五年中,慢病毒载体已发展成为向神经系统输送基因的极其有效的工具,为治疗此类疾病揭示了广泛的可能性。这篇综述描述了慢病毒载体开发过程中最重要和最新的进展,以及在人类神经退行性疾病动物模型中的原理验证。