Gene therapy for amyotrophic lateral sclerosis

被引：15
|
作者：
Federici, Thais [1 ]
Boulis, Nicholas M. [1 ]
机构：
[1] Emory Univ, Dept Neurosurg, Atlanta, GA 30322 USA
来源：
NEUROBIOLOGY OF DISEASE | 2012年 / 48卷 / 02期
关键词：
Astrocytes; Gene delivery; Motor neurons; Neurotrophic factors; RNA interference; Superoxide dismutase 1; PLURIPOTENT STEM-CELLS; GLUTAMATE TRANSPORTER EAAT2; MOTOR-NEURON DEGENERATION; MOUSE MODEL; RAT MODEL; DISEASE PROGRESSION; PROLONGS SURVIVAL; RNA INTERFERENCE; TRANSGENIC MODEL; DELIVERY;
D O I：
10.1016/j.nbd.2011.08.018
中图分类号：
Q189 [神经科学];
学科分类号：
071006 ;
摘要：
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. (C) 2011 Elsevier Inc. All rights reserved.
引用
收藏
页码：236 / 242
页数：7
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