Novel cell therapy for Hirschsprung Disease: from patient iPS cells to large animal trials

先天性巨结肠症的新型细胞疗法：从患者 iPS 细胞到大型动物试验

• 批准号: nhmrc : GNT1125805
• 负责人: A/Pr Sebastian King
• 金额: $ 101.18万
• 依托单位国家: 澳大利亚
• 项目类别: Project Grants
• 财政年份: 2017
• 资助国家: 澳大利亚
• 起止时间: 2017-01-01 至 无数据
• 项目状态: 已结题
• 来源: https://researchdata.edu.au/novel-cell-therapy-animal-trials/1347513
• 关键词: Novel; cell; therapy; Hirschsprung; Disease

In Hirschsprung disease the lower bowel has no nerve cells. It does not function so it is surgically removed but quality of life is poor. A new idea is to replace the missing cells with new ones. Human infants are very large so we will use new stem cell technologies to create large numbers of cells. We will use polymer chemistry to devise a method of getting the cells into such a large organ as the bowel, and trial these on a model, the piglet, which closely resembles in size the human baby.

先天性巨结肠病的下肠没有神经细胞。它不起作用，所以要通过手术切除，但生活质量很差。一个新的想法是用新的细胞替换缺失的细胞。人类婴儿非常大，所以我们将使用新的干细胞技术来制造大量的细胞。我们将使用聚合物化学来设计一种方法，将细胞植入像肠道这样的大器官，并在模型仔猪上进行试验，它的大小与人类婴儿非常相似。