用于脊髓神经变性的干细胞移植。
Stem cell transplantation for spinal cord neurodegeneration.
作者信息
Lepore Angelo C, Maragakis Nicholas J
机构信息
Department of Neurology, The Johns Hopkins University School of Medicine, Baltimore, MD, USA.
出版信息
Methods Mol Biol. 2011;793:479-93. doi: 10.1007/978-1-61779-328-8_31.
Amyotrophic lateral sclerosis (ALS), a disorder that affects 30,000 individuals in the USA alone, is characterized by relatively rapid degeneration of upper and lower motor neurons, with death normally occurring 2-5 years following diagnosis due to respiratory paralysis. Transplantation of various classes of neural precursor cells (NPCs) is a promising therapeutic strategy for the treatment of traumatic CNS injury and neurodegeneration, including ALS, because of the ability to replace lost or dysfunctional CNS cell types, provide neuroprotection, and deliver gene factors of interest. In order to target cellular therapy to diaphragmatic dysfunction in models of ALS, NPCs can be transplanted specifically into the cervical spinal cord ventral gray matter of both SOD1G93A rats and mice. The SOD1G93A rats and mice are currently the most well-studied animal model of the disease.
肌萎缩侧索硬化症（ALS）仅在美国就影响着3万人，其特征是上下运动神经元相对快速退化，通常在诊断后2至5年因呼吸麻痹而死亡。由于能够替代丢失或功能失调的中枢神经系统细胞类型、提供神经保护并传递感兴趣的基因因子，各类神经前体细胞（NPC）移植是治疗创伤性中枢神经系统损伤和神经退行性疾病（包括ALS）的一种有前景的治疗策略。为了在ALS模型中将细胞疗法靶向膈肌功能障碍，可以将NPC特异性移植到SOD1G93A大鼠和小鼠的颈髓腹侧灰质中。SOD1G93A大鼠和小鼠是目前对该疾病研究最为深入的动物模型。
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