Induced pluripotent stem cell therapy for lipodystrophy

诱导多能干细胞治疗脂肪营养不良

基本信息

批准号：
8542832
负责人：
SUSAN M MAJKA
金额：
$ 18.82万
依托单位：
VANDERBILT UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
2012
资助国家：
美国
起止时间：
2012-09-15 至 2015-08-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8542832
关键词：
Address Adipocytes Adipose tissue Adult Age Animal Model Autoimmunity Autologous Blood Glucose Bone Marrow Cartilage Cell Therapy Cell model Cells Clinic Communities Defect Development Diabetes Mellitus Disease Dyslipidemias Engraftment Environment Fatty Liver Fatty acid glycerol esters Fibroblasts Foundations Functional disorder Future Gene Targeting Genes Genetic Graft Survival HIV Histology Human Hypertriglyceridemia In Vitro Individual Inflammation Inherited Insulin Resistance Laboratories Length Lipids Lipodystrophy Liver Medical Mesenchymal Mesenchymal Stem Cells Messenger RNA Metabolic Methods Mitochondria Modeling Modification Molecular Mouse Strains Mus Mutation National Institute of Diabetes and Digestive and Kidney Diseases Natural regeneration Non-Insulin-Dependent Diabetes Mellitus Patients Peptide Hydrolases Phenotype Reagent Relative (related person)Research Resources Sampling Site Skeletal Muscle Skin Somatic Cell Source Technology Testing Therapeutic Therapeutic Uses Tissues Transfection Transgenes Transplantation Viral Vector Work Zinc Fingers adipocyte differentiation aged blood glucose regulation bone c-myc Genes cell type embryonic stem cell gene correction gene therapy improved functioning in vivo induced pluripotent stem cell innovation interest lipid biosynthesis mouse model nuclease public health relevance repaired self-renewal senescence stem cell therapy telomere therapy development tissue regeneration tissue repair transcription factor tumor

项目摘要

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.

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，我们假设小鼠 iPS 细胞衍生的间充质干细胞和脂肪细胞为治疗脂肪营养不良提供基于细胞的疗法的潜力。我们建议使用无转基因方法创建一组脂肪营养不良的小鼠 iPS 细胞模型，目的是评估年轻与老年衍生 iPS 相对于 BM-MSC 向间充质和脂肪谱系及后续功能的分化潜力。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，这些研究将产生科学界和医学界都非常感兴趣的重要资源和试剂。