有哪些信誉好的足球投注网站generation of functional neutrophils from a mouse model of x-linked chronic granulomatous disorder using induced pluripotent stem cells代的中性粒细胞功能的小鼠模型和x染色体相关慢性肉芽肿性疾病使用诱导多能干细胞.pdfVIP
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generation of functional neutrophils from a mouse model of x-linked chronic granulomatous disorder using induced pluripotent stem cells代的中性粒细胞功能的小鼠模型和x染色体相关慢性肉芽肿性疾病使用诱导多能干细胞
Generation of Functional Neutrophils from a Mouse Model of X-Linked Chronic Granulomatous Disorder Using Induced Pluripotent Stem Cells 1 1 1 2 2 Sayandip Mukherjee , Giorgia Santilli , Michael P. Blundell , Susana Navarro , Juan A. Bueren , Adrian J. Thrasher1,3* 1 Centre for Immunodeficiency, UCL Institute of Child Health, London, United Kingdom, 2 Hematopoiesis and Gene Therapy Division, Centro de Investigaciones ´ ´ Energeticas, Medioambientales y Tecnologicas (CIEMAT), Madrid, Spain, 3 Great Ormond Street Hospital NHS Trust, London, United Kingdom Abstract Murine models of human genetic disorders provide a valuable tool for investigating the scope for application of induced pluripotent stem cells (iPSC). Here we present a proof-of-concept study to demonstrate generation of iPSC from a mouse model of X-linked chronic granulomatous disease (X-CGD), and their successful differentiation into haematopoietic progenitors of the myeloid lineage. We further demonstrate that additive gene transfer using lentiviral vectors encoding gp91phox is capable of restoring NADPH-oxidase activity in mature neutrophils derived from X-CGD iPSC. In the longer term, correction of iPSC from human patients with CGD has therapeutic potential not only through generation of transplantable haematopoietic stem cells, but also through production of large numbers of autologous functional neutrophils. Citation: Mukherjee S, Santilli G, Blundell MP, Navarro S, Bueren JA, et al. (2011) Generation of Functional Neutrophils from a Mouse Model of X-Linked Chronic Granulomatous Disorder Using
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