Manufacturing of iPS cell derived neurons from patients with familial Alzhiemer's disease for drug discovery and stratified medicine applications

由于缺乏合适的体外模型，特别是对于难以获得或获得受影响细胞类型的疾病，阻碍了对许多疾病的理解。诱导多能干细胞（iPSC）是一种理想的替代来源，因为它们可以来自患者的皮肤细胞，并有潜力在体内形成任何细胞类型。然而，目前的技术受到无法可靠地产生成熟细胞类型的阻碍。我们已经开发出一种方法，使iPSC能够以低成本和高纯度可靠地分化为神经元。这提供了利用这些细胞在培养皿中了解阿尔茨海默病（AD）并为此类患者开发新的治疗和诊断方法的能力。最终，我们的目标是开发一种能够诊断AD并为患者提供治疗方案的技术，并为行业提供筛选和开发这种不治之症新药的能力。

Progress in understanding many diseases has been hindered by a lack of suitable in vitro models, particularly for diseases in which affected cell types are difficult to access or obtain. Induced pluripotent stem cells (iPSCs) are an ideal alternative source as they can be derived from patients' skin cells and have the potential to form any cell type in the body. However, current technologies are hindered by the inability to reliably produce mature cell types. We have developed a method that enables iPSCs to be reliably differentiated at low cost and high purity into neurons. This offers the ability to utilise these cells for understanding Alzheimer's disease (AD) in the petri dish and developing new treatments and diagnostics for such patients. Ultimately, we aim to produce a technology capable of diagnosing AD and informing treatment options for patients as well as providing industry with the ability to screen and develop novel drugs for this incurable disease.