The NCLs (neuronal ceroid lipofuscinosis) are forms of neurodegenerative disease that affect people of all ages and ethnicities but are most prevalent in children. Commonly known as Batten disease, this debilitating neurological disorder is comprised of 13 different subtypes that are categorized based on the particular gene that is mutated (CLN1-8, CLN10-14). The pathological mechanisms underlying the NCLs are not well understood due to our poor understanding of the functions of NCL proteins. Only one specific treatment (enzyme replacement therapy) is approved, which is for the treating the brain in CLN2 disease. Hence there remains a desperate need for further research into disease-modifying treatments. In this review, we present and evaluate the genes, proteins and studies performed in the social amoeba, nematode, fruit fly, zebrafish, mouse and large animals pertinent to NCL. In particular, we highlight the use of multicellular model organisms to study NCL protein function, pathology and pathomechanisms. Their use in testing novel therapeutic approaches is also presented. With this information, we highlight how future research in these systems may be able to provide new insight into NCL protein functions in human cells and aid in the development of new therapies.

中文翻译：

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多细胞模型生物对神经元类脂褐藻病研究的贡献。

NCL（神经性类固醇脂褐藻病）是神经退行性疾病的一种形式，会影响所有年龄和种族的人，但在儿童中最为普遍。通常称为巴滕病，这种使人衰弱的神经疾病由13种不同的亚型组成，这些亚型是根据突变的特定基因（CLN1-8，CLN10-14）进行分类的。由于我们对NCL蛋白质功能的了解不足，因此尚未很好地理解NCL的病理机制。仅批准了一种特定的治疗方法（酶替代疗法），用于治疗CLN2疾病的大脑。因此，迫切需要对疾病缓解疗法进行进一步研究。在这篇评论中，我们介绍并评估了在变形虫，线虫，果蝇，斑马鱼，与NCL有关的小鼠和大型动物。特别是，我们着重介绍了使用多细胞模型生物来研究NCL蛋白功能，病理和致病机理。还介绍了它们在测试新型治疗方法中的用途。利用这些信息，我们强调了这些系统中的未来研究如何能够提供对NCL蛋白质在人细胞中功能的新见解，并有助于开发新疗法。