The Use of Patient-Specific Induced Pluripotent Stem Cells (iPSCs) to Identify Osteoclast Defects in Rare Genetic Bone Disorders.
The Use of Patient-Specific Induced Pluripotent Stem Cells (iPSCs) to Identify Osteoclast Defects in Rare Genetic Bone Disorders.
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DOI：
10.3390/jcm3041490
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发表时间：
2014-12-17
影响因子：
3.9
通讯作者：
Chen IP
中科院分区：
文献类型：
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作者：
Chen IP
已经描述了500多种罕见的遗传性骨病，但对其中许多人来说，可用的治疗选择有限。研究这些骨骼疾病的挑战来自于缺乏合适的动物模型和无法获得用于研究的骨骼组织。骨病的骨骼异常的影响因素可能是成骨细胞引导的异常骨形成或破骨细胞的异常骨吸收，或两者兼而有之。患者特异性诱导多能干细胞(IPSCs)可以从不同组织来源的体细胞中产生，理论上可以分化为任何所需的细胞类型。然而，成功地将HiPSCs分化为有功能的骨细胞仍然是一个挑战。我们小组的重点是使用人ipscs(HiPSCs)来鉴定颅骨干骺端发育不良患者的破骨细胞缺陷。在这篇综述中，我们描述了干细胞技术在更好地治疗这类疾病的研究中的影响，罕见遗传性骨病患者的hPSCs的产生，以及目前将hPSCs分化为破骨细胞的方案。
More than 500 rare genetic bone disorders have been described, but for many of them only limited treatment options are available. Challenges for studying these bone diseases come from a lack of suitable animal models and unavailability of skeletal tissues for studies. Effectors for skeletal abnormalities of bone disorders may be abnormal bone formation directed by osteoblasts or anomalous bone resorption by osteoclasts, or both. Patient-specific induced pluripotent stem cells (iPSCs) can be generated from somatic cells of various tissue sources and in theory can be differentiated into any desired cell type. However, successful differentiation of hiPSCs into functional bone cells is still a challenge. Our group focuses on the use of human iPSCs (hiPSCs) to identify osteoclast defects in craniometaphyseal dysplasia. In this review, we describe the impact of stem cell technology on research for better treatment of such disorders, the generation of hiPSCs from patients with rare genetic bone disorders and current protocols for differentiating hiPSCs into osteoclasts.