Emory University, Department of Neurosurgery, 1365B Clifton Road NE, Suite B6200, Atlanta, GA 30322, USA.
Neurobiol Dis. 2012 Nov;48(2):236-42. doi: 10.1016/j.nbd.2011.08.018. Epub 2011 Aug 25.
Gene therapy continues to be a potential option for amyotrophic lateral sclerosis (ALS). This chapter will inform the reader about promising therapeutic transgenes and proof-of-principle studies in transgenic rodent models of ALS. Challenges regarding the disease targets and time for therapeutic intervention will be also discussed. Finally, restorative therapy for ALS, as well as gene therapy for other motor neuron diseases will be briefly reviewed.
基因治疗仍然是肌萎缩侧索硬化症（ALS）的一种潜在选择。本章将向读者介绍有前途的治疗性转基因和 ALS 转基因啮齿动物模型中的原理验证研究。还将讨论有关疾病靶标和治疗干预时间的挑战。最后，简要回顾 ALS 的修复治疗以及其他运动神经元疾病的基因治疗。
