运动神经元疾病的细胞疗法

Cellular therapies in motor neuron diseases.

作者信息

Nayak Mamatha S, Kim Yun-Sook, Goldman Michelle, Keirstead Hans S, Kerr Douglas A

机构信息

Department of Neurology, Johns Hopkins University School of Medicine, 600 North Wolfe Street, Baltimore, MD 21287, USA.

出版信息

Biochim Biophys Acta. 2006 Nov-Dec;1762(11-12):1128-38. doi: 10.1016/j.bbadis.2006.06.004. Epub 2006 Jun 17.

DOI:10.1016/j.bbadis.2006.06.004

PMID:16872810

Abstract

Amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA) are prototypical motor neuron diseases that result in progressive weakness as a result of motor neuron dysfunction and death. Though much work has been done in both diseases to identify the cellular mechanisms of motor neuron dysfunction, once motor neurons have died, one of potential therapies to restore function would be through the use of cellular transplantation. In this review, we discuss potential strategies whereby cellular therapies, including the use of stem cells, neural progenitors and cells engineered to secrete trophic factors, may be used in motor neuron diseases. We review pre-clinical data in rodents with each of these approaches and discuss advances and regulatory issues regarding the use of cellular therapies in human motor neuron diseases.

摘要

肌萎缩侧索硬化症（ALS）和脊髓性肌萎缩症（SMA）是典型的运动神经元疾病，由于运动神经元功能障碍和死亡导致进行性肌无力。尽管在这两种疾病中已经开展了大量工作来确定运动神经元功能障碍的细胞机制，但一旦运动神经元死亡，恢复功能的一种潜在疗法可能是通过细胞移植。在本综述中，我们讨论了细胞疗法的潜在策略，包括使用干细胞、神经祖细胞和经基因工程改造以分泌神经营养因子的细胞，这些疗法可用于治疗运动神经元疾病。我们回顾了采用上述每种方法在啮齿动物中开展的临床前数据，并讨论了在人类运动神经元疾病中使用细胞疗法的进展和监管问题。