Therapeutic opportunities and challenges of induced pluripotent stem cells-derived motor neurons for treatment of amyotrophic lateral sclerosis and motor neuron disease

查看全文 作　　者：Manoj Kumar [1,2]Jaiswal

机构地区：[1]Molecular Imaging and Neuropathology Division, New York State Psychiatry Institute, Columbia University, New York, NY, USA;[2]Department of Psychiatry, Columbia University, New York, NY, USA

出　　处：《Neural Regeneration Research》索引2017年第12卷第5期,共14页

摘　　要：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 in vitro 'disease in dish' 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. 关键词：IPSCS stem cells human patients ALS MITOCHONDRIA motor neuron disease disease modeling NEURODEGENERATION gene editing transplantation drug screening

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Therapeutic opportunities and challenges of induced pluripotent stem cells-derived motor neurons for treatment of amyotrophic lateral sclerosis and motor neuron disease

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