Regenerative Medicine Institute, Department of Biomedical Sciences, Cedars-Sinai Medical Center, Los Angeles, CA 90048, USA.
Neurotherapeutics. 2011 Oct;8(4):591-606. doi: 10.1007/s13311-011-0068-7.
Motor neuron degeneration leading to muscle atrophy and death is a pathological hallmark of disorders, such as amyotrophic lateral sclerosis or spinal muscular atrophy. No effective treatment is available for these devastating diseases. At present, cell-based therapies targeting motor neuron replacement, support, or as a vehicle for the delivery of neuroprotective molecules are being investigated. Although many challenges and questions remain, the beneficial effects observed following transplantation therapy in animal models of motor neuron disease has sparked hope and a number of clinical trials. Here, we provide a comprehensive review of cell-based therapeutics for motor neuron disorders, with a particular emphasis on amyotrophic lateral sclerosis.
运动神经元退化导致肌肉萎缩和死亡是肌萎缩侧索硬化症或脊髓性肌萎缩症等疾病的病理标志。目前，针对运动神经元替代、支持或作为神经保护分子传递载体的细胞治疗方法正在研究中。尽管存在许多挑战和问题，但运动神经元疾病动物模型中移植治疗观察到的有益效果激发了希望并开展了多项临床试验。在这里，我们全面综述了运动神经元疾病的基于细胞的治疗方法，特别强调肌萎缩侧索硬化症。
