Characterising the molecular basis of cystic kidney diseases using kidney organoids created by directed differentiation of patient-derived, induced pluripotent stem cells.

遗传基因突变导致了儿童近一半的慢性肾脏疾病。在大多数情况下，我们不知道这种突变是什么，也不知道它是如何导致肾脏疾病的。在这项研究中，我们将把患有肾脏疾病的儿童的皮肤细胞转化为干细胞，然后用这些干细胞在盘子里制造一个迷你肾脏。这将作为肾脏疾病的模型，使我们能够了解细胞内变化水平的问题是什么。这可能会带来治疗肾脏疾病的新方法。

Inherited genetic mutations cause almost half of chronic kidney diseases in children. In most cases we do not know what the mutation is or how it causes kidney disease. In this study we will turn skin cells from children with kidney disease into stem cells and then use these to make a mini-kidney in a dish. This will act as a model of kidney disease allowing us to understand what the problem is at the level of changes within the cells. This may result in new ways of treating kidney disease.