Disruption to intracellular trafficking as a central pathogenic mechanism in amyotrophic lateral sclerosis (ALS)

ALS （MND）有几种不同的形式，但疾病在症状和病理方面看起来非常相似。我们已经确定了几种不同形式的ALS共有的共同疾病过程，这表明这是运动神经元死亡的潜在机制。本研究将探讨是否可以在动物疾病模型中基于这一机制开发新的药物靶点。这可能最终有助于开发新的ALS治疗方法。

There are several different forms of ALS (MND), but the disease appears very similar in terms of symptoms and pathology. We have identified a common disease process shared by several different forms of ALS, which suggests that this is the underlying mechanism by which motor neurons die. This study will investigate whether we can develop new drug targets based on this mechanism in animal disease models. This may ultimately assist in the development of new treatments for ALS.