Amyotrophic lateral sclerosis (ALS) is a rapidly progressive and ultimately fatal neurodegenerative disorder of unknown aetiology that involves the loss of upper and lower motor neurons in the cerebral cortex, brainstem and spinal cord. Significant progress in understanding the cellular mechanisms of motor neuron degeneration in ALS has not been matched with the development of therapeutic strategies to prevent disease progression, and riluzole remains the only available therapy, with only marginal effects on disease survival. More recently alterations of mRNA processing in genetically defined forms of ALS, as those related to TDP-43 and FUS-TLS gene mutations have provided important insights into the molecular networks implicated in the disease pathogenesis. Here we review some of the recent progress in promoting therapeutic strategies for neurodegeneration.

中文翻译：

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肌萎缩性侧索硬化症神经退行性变的临床方法策略。

肌萎缩性侧索硬化症（ALS）是一种病因不明的快速进行性，最终致命的神经退行性疾病，涉及大脑皮质，脑干和脊髓中上，下运动神经元的丢失。在了解ALS中运动神经元变性的细胞机制方面的重大进展尚未与预防疾病进展的治疗策略的发展相提并论，利鲁唑仍然是唯一可用的治疗方法，对疾病的存活率仅有很小的影响。最近，与TDP-43和FUS-TLS基因突变相关的遗传修饰形式的ALS中的mRNA处理改变，为深入研究与疾病发病机理有关的分子网络提供了重要见识。