干细胞技术在运动神经元疾病研究和治疗中的应用。
Stem cell technology for the study and treatment of motor neuron diseases.
机构信息
University of Michigan Department of Neurology, 109 Zina Pitcher Place, Ann Arbor, MI 48109, USA.
出版信息
Regen Med. 2011 Mar;6(2):201-13. doi: 10.2217/rme.11.6.
Abstract
Amyotrophic lateral sclerosis and spinal muscular atrophy are devastating neurodegenerative diseases that lead to the specific loss of motor neurons. Recently, stem cell technologies have been developed for the investigation and treatment of both diseases. Here we discuss the different stem cells currently being studied for mechanistic discovery and therapeutic development, including embryonic, adult and induced pluripotent stem cells. We also present supporting evidence for the utilization of stem cell technology in the treatment of amyotrophic lateral sclerosis and spinal muscular atrophy, and describe key issues that must be considered for the transition of stem cell therapies for motor neuron diseases from bench to bedside. Finally, we discuss the first-in-human Phase I trial currently underway examining the safety and feasibility of intraspinal stem cell injections in amyotrophic lateral sclerosis patients as a foundation for translating stem cell therapies for various neurological diseases.
摘要
肌萎缩侧索硬化症和脊髓性肌萎缩症是两种具有破坏性的神经退行性疾病，会导致运动神经元的特异性丧失。最近，干细胞技术已经被开发出来，用于这两种疾病的研究和治疗。在这里，我们讨论了目前正在研究的不同干细胞，用于机制发现和治疗开发，包括胚胎干细胞、成体干细胞和诱导多能干细胞。我们还提供了支持使用干细胞技术治疗肌萎缩侧索硬化症和脊髓性肌萎缩症的证据，并描述了将运动神经元疾病的干细胞疗法从实验室转化到临床应用必须考虑的关键问题。最后，我们讨论了目前正在进行的首例人体 I 期临床试验，该试验旨在评估脊髓内干细胞注射治疗肌萎缩侧索硬化症患者的安全性和可行性，为各种神经疾病的干细胞疗法转化奠定基础。
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