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来扩大iPS的使用，以改变NIDDK相关疾病的治疗模式。因此，我们假设，鼠iPS细胞衍生的间充质干细胞和脂肪细胞提供了基于细胞的疗法来治疗脂肪营养不良的潜力。我们建议使用无转基因方法建立一组脂肪营养不良的小鼠iPS细胞模型，以评估年轻与老年衍生的iPS相对于BM-MSC向间充质和脂肪谱系的分化潜力以及随后的功能。我们还将测试iPS细胞衍生的间充质干细胞相对于BM-MSC拯救脂肪营养不良小鼠模型的能力。该模型将使我们能够识别可能适合作为潜在疗法的细胞类型。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.