Modeling Kidney Disease with iPS Cells.
Modeling Kidney Disease with iPS Cells.
复制标题
用IPS细胞对肾脏疾病进行建模。
DOI：
10.4137/bmi.s20054
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发表时间：
2015
影响因子：
3.8
通讯作者：
Freedman BS
中科院分区：
文献类型：
--
作者：
Freedman BS
诱导的多能干细胞（IPSC）是已转录到胚胎干细胞（ESC）状态的躯体细胞。 ITIS，目标在IPSC中产生替代组织和“疾病”。型号肾毒性化学损伤在定向分化和CRISPR基因组编辑方面的进展可以使IPSC模型更具特定的IPSC模型，并为诊断，疾病建模，治疗筛查和使用人类细胞的组织再生提供了新的可能性。
Induced pluripotent stem cells (iPSCs) are somatic cells that have been transcriptionally reprogrammed to an embryonic stem cell (ESC)-like state. iPSCs are a renewable source of diverse somatic cell types and tissues matching the original patient, including nephron-like kidney organoids. iPSCs have been derived representing several kidney disorders, such as ADPKD, ARPKD, Alport syndrome, and lupus nephritis, with the goals of generating replacement tissue and ‘disease in a dish’ laboratory models. Cellular defects in iPSCs and derived kidney organoids provide functional, personalized biomarkers, which can be correlated with genetic and clinical information. In proof of principle, disease-specific phenotypes have been described in iPSCs and ESCs with mutations linked to polycystic kidney disease or focal segmental glomerulosclerosis. In addition, these cells can be used to model nephrotoxic chemical injury. Recent advances in directed differentiation and CRISPR genome editing enable more specific iPSC models and present new possibilities for diagnostics, disease modeling, therapeutic screens, and tissue regeneration using human cells. This review outlines growth opportunities and design strategies for this rapidly expanding and evolving field.