Induced pluripotent stem cell therapy for lipodystrophy

描述（由申请人提供）：拟议的研究将使用创新方法扩大iPS的使用，以改变niddk相关疾病的治疗范例。因此，我们假设小鼠iPS细胞衍生的间充质干细胞和脂肪细胞为细胞治疗脂肪营养不良提供了潜力。我们建议使用无转基因方法创建一组小鼠脂肪营养不良的iPS细胞模型，目的是评估相对于BM-MSC到间充质和脂肪谱系的年轻和年老的iPS分化潜力和随后的功能。我们还将测试iPS细胞衍生的间充质干细胞拯救与BM-MSC相关的脂肪营养不良小鼠模型的能力。这个模型将使我们能够确定一种可能适合作为潜在治疗方法的细胞类型。此外，这些研究将产生重要的资源和试剂，这将是科学界和医学界的巨大兴趣。

DESCRIPTION (provided by applicant): The proposed studies will use innovative approaches to expand the use of iPS to change treatment paradigms for NIDDK-related diseases. We therefore hypothesize that murine iPS cell derived mesenchymal stem cells and adipocytes offer the potential for cell-based therapy to treat lipodystrophy. We propose to create a set of mouse iPS cell models of lipodystrophy using transgene-free approaches for the purpose of evaluating the differentiation potential of young versus aged derived iPS relative to BM-MSC to mesenchymal and adipose lineages and subsequent function. We will also test the ability of the iPS cell derived mesenchymal stem cells to rescue a mouse model of lipodystrophy relative to BM-MSC. This model will enable us to identify a cell type which may be suited as a potential therapy. Additionally, these studies will generate important resources and reagents that will be of vast interest to both the scientific and medical communities.