Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.
Generation of functional neutrophils from a mouse model of X-linked chronic granulomatous disorder using induced pluripotent stem cells.
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DOI：
10.1371/journal.pone.0017565
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发表时间：
2011-03-03
期刊：
影响因子：
3.7
通讯作者：
Thrasher AJ
中科院分区：
文献类型：
--
作者：
Mukherjee S;Santilli G;Blundell MP;Navarro S;Bueren JA;Thrasher AJ
人类遗传疾病的小鼠模型为研究诱导多能干细胞（iPSC）的应用范围提供了有价值的工具。在这里，我们提出了一个概念验证研究，以证明从X连锁慢性肉芽肿病（X-CGD）的小鼠模型中产生iPSC，并成功分化为骨髓系的造血祖细胞。我们进一步证明了使用编码gp 91 phox的慢病毒载体的加性基因转移能够恢复来自X-CGD iPSC的成熟中性粒细胞中的NADPH氧化酶活性。从长远来看，从患有CGD的人类患者中校正iPSC不仅通过产生可移植的造血干细胞，而且通过产生大量自体功能性中性粒细胞具有治疗潜力。
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.
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影响因子：
64.5
作者：
Park IH;Arora N;Huo H;Maherali N;Ahfeldt T;Shimamura A;Lensch MW;Cowan C;Hochedlinger K;Daley GQ
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作者：
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