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Cord blood as a potential therapeutic for amyotrophic lateral sclerosis

机译:脐带血作为肌营养侧面硬化的潜在治疗

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Introduction: Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disease characterized by progressive motor neuron degeneration in the brain and spinal cord. Treatment options are limited due to the complexity of underlying disease factors. Cell therapy, using human umbilical cord blood (hUCB) cells may be a promising new treatment for ALS, mainly by providing a protective microenvironment for motor neuron survival.Areas covered: Composition, in vitro and in vivo differentiation of hUCB cells, and the advantages of cord blood as a source of transplant cells are discussed. A brief history of hUCB in treatment of an ALS animal model and the feasibility of these cells in therapy for ALS patients is provided. Current ALS clinical trials are also deliberated.Expert opinion: Among multiple advantages, hUCB cells' production of various anti-inflammatory/growth/trophic factors makes them an attractive cell source for ALS therapy. Biodistribution and optimal hUCB cell dose for transplantation have been determined in preclinical studies. Repeated intravenous cell doses during disease progression may be the best approach for cell-based ALS treatment. Accumulated evidence shows the efficacy of naive or genetically modified MNC hUCB cells in the treatment of ALS and provide a superior basis for the development of clinical trials in the near future.
机译:介绍:肌萎缩侧面硬化症(ALS)是一种致命的神经变性疾病,其特征在于脑和脊髓的渐进式电机神经元变性。由于潜在的疾病因素的复杂性,治疗方案受到限制。细胞疗法,使用人的脐带血(HUCB)细胞可能是ALS的有希望的新处理,主要是为运动神经元存活的保护性微环境。覆盖:组合物,体外和Hucb细胞的体内分化,以及优势讨论了作为移植细胞来源的脐带血。提供了HUCB治疗ALS动物模型的历史和对ALS患者治疗中这些细胞的可行性。目前的ALS临床试验也是审议的。普及意见:在多种优势中,HUCB细胞的各种抗炎/生长/营养因子的产生使其成为ALS治疗的有吸引力的细胞来源。在临床前研究中确定了用于移植的生物分布和最佳HUCB细胞剂量。在疾病进展期间重复静脉内细胞剂量可能是用于细胞的ALS治疗的最佳方法。累积的证据显示幼稚或遗传修饰的MNC HUCB细胞在治疗ALS中的疗效,并为在不久的将来提供临床试验的优越依据。

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