Baekelandt V, De Strooper B, Nuttin B, Debyser Z
Laboratory for Experimental Neurosurgery and Neuroanatomy, CEHA Provisorium 1, Minderbroedersstraat 17, B-3000 Leuven, Belgium.
Curr Opin Mol Ther. 2000 Oct;2(5):540-54.
Gene transfer into the central nervous system by ex vivo or in vivo techniques is a rapidly emerging field in neuroscience. Potential applications of gene therapy for the nervous system include not only congenital single gene disorders, but also brain tumors and acquired chronic diseases. Considerable progress has been made in the understanding of neurodegenerative diseases such as Parkinson's and Alzheimer's disease. As a result, gene therapy for Parkinson's, and possibly Alzheimer's disease could be regarded as a realistic alternative to the limited treatment options currently available. In this review, we highlight the most important developments in gene transfer techniques as well as the newest insights in the mechanisms of some neurodegenerative disorders and put these into the perspective of gene therapeutic strategies for the central nervous system.
通过离体或体内技术将基因导入中枢神经系统是神经科学中一个迅速兴起的领域。基因治疗在神经系统的潜在应用不仅包括先天性单基因疾病，还包括脑肿瘤和后天性慢性疾病。在对帕金森病和阿尔茨海默病等神经退行性疾病的理解方面已经取得了相当大的进展。因此，帕金森病以及可能的阿尔茨海默病的基因治疗可以被视为目前有限治疗选择的一种现实替代方案。在这篇综述中，我们重点介绍了基因转移技术的最重要进展以及一些神经退行性疾病机制的最新见解，并将这些置于中枢神经系统基因治疗策略的背景下进行考量。
