Jaiswal Manoj Kumar
Molecular Imaging and Neuropathology Division, New York State Psychiatry Institute, Columbia University, New York, NY, USA.
Department of Psychiatry, Columbia University, New York, NY, USA.
Neural Regen Res. 2017 May;12(5):723-736. doi: 10.4103/1673-5374.206635.
Amyotrophic lateral sclerosis (ALS) and motor neuron diseases (MNDs) are progressive neurodegenerative diseases that affect nerve cells in the brain affecting upper and lower motor neurons (UMNs/LMNs), brain stem and spinal cord. The clinical phenotype is characterized by loss of motor neurons (MNs), muscular weakness and atrophy eventually leading to paralysis and death due to respiratory failure within 3-5 years after disease onset. No effective treatment or cure is currently available that halts or reverses ALS and MND except FDA approved drug riluzole that only modestly slows the progression of ALS in some patients. Recent advances in human derived induced pluripotent stem cells have made it possible for the first time to obtain substantial amounts of human cells to recapitulate "" and test some of the underlying pathogenetic mechanisms involved in ALS and MNDs. In this review, I discussed the opportunities and challenges of induced pluropotent stem cells-derived motor neurons for treatment of ALS and MND patients with special emphasis on their implications in finding a cure for ALS and MNDs.
肌萎缩侧索硬化症（ALS）和运动神经元疾病（MNDs）是进行性神经退行性疾病，会影响大脑中的神经细胞，累及上、下运动神经元（UMNs/LMNs）、脑干和脊髓。临床表型的特征是运动神经元（MNs）丧失、肌肉无力和萎缩，最终导致瘫痪，并在疾病发作后3至5年内因呼吸衰竭而死亡。目前尚无有效的治疗方法或治愈手段能够阻止或逆转ALS和MND，除了美国食品药品监督管理局（FDA）批准的药物利鲁唑，它仅能在一些患者中适度减缓ALS的进展。人类诱导多能干细胞的最新进展首次使得获取大量人类细胞以重现疾病情况并测试一些与ALS和MNDs相关的潜在致病机制成为可能。在这篇综述中，我讨论了诱导多能干细胞衍生的运动神经元用于治疗ALS和MND患者的机遇与挑战，并特别强调了它们在寻找ALS和MNDs治愈方法方面的意义。
