University of North Carolina Gene Therapy Center, University of North Carolina at Chapel Hill, Chapel Hill, NC, United States.
Exp Neurol. 2013 Jun;244:27-35. doi: 10.1016/j.expneurol.2011.10.003. Epub 2011 Oct 8.
This review addresses the state of gene therapy research for the treatment of epilepsy. Preclinical studies have demonstrated the anti-seizure efficacy of viral vector-based gene transfer through the use of a variety of strategies - from modulating classic neurotransmitter systems to targeting or overexpressing of neuropeptide receptors in seizure-specific brain regions. While these studies provide substantive proof of principle for viral vector gene therapy, future studies must address the challenges of vector immunity, cellular specificity and effective global delivery. As these issues are resolved, viral vector gene therapy should significantly impact the treatment of intractable epilepsy.
本综述探讨了基因治疗癫痫的研究现状。通过使用多种策略，包括调节经典神经递质系统，靶向或过表达致痫脑区的神经肽受体，病毒载体的基因转移已在临床前研究中证实了其抗癫痫作用。这些研究为病毒载体基因治疗提供了实质性的原理证明，但未来的研究必须解决载体免疫、细胞特异性和有效全局传递等问题。随着这些问题的解决，病毒载体基因治疗应该会对治疗难治性癫痫产生重大影响。
