Neurodegenerative Diseases such as Alzheimer’s Disease represent a major public health challenge, with no disease modifying therapies available. The availability of induced pluripotent stem cells from patients with phenotypes and genotypes of interest, that can be subsequently differentiated in vitro into disease-affected cell types, has revolutionised our ability to generate physiologically relevant disease models. The recent availability of brain organoids – self-organising in vitro tissue models – as enabled the generation of complex, multicellular systems to study brain development and disease. Although widely used for modelling neurodevelopment, early studies have demonstrated great promise in the use of organoids as models of neurodegenerative disease. Here, I will review recent progress to model neurodegenerative diseases using organoids and comment on future directions and challenges.

阿尔茨海默病等神经退行性疾病是一项重大的公共卫生挑战，目前尚无可用的疾病改善疗法。来自具有感兴趣表型和基因型的患者的诱导多能干细胞的可用性，随后可以在体外分化成受疾病影响的细胞类型，这彻底改变了我们生成生理相关疾病模型的能力。最近出现的脑类器官——体外自组织组织模型——能够生成复杂的多细胞系统来研究大脑发育和疾病。虽然广泛用于模拟神经发育，但早期研究表明，在使用类器官作为神经退行性疾病模型方面具有很大的前景。在这里，我将回顾使用类器官模拟神经退行性疾病的最新进展，并评论未来的方向和挑战。