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本文引用的文献

1
iPSC-derived β cells model diabetes due to glucokinase deficiency.iPSC 分化的β细胞可模拟因葡萄糖激酶缺乏所致的糖尿病。
J Clin Invest. 2013 Jul;123(7):3146-53. doi: 10.1172/JCI67638. Epub 2013 Jun 17.
2
Modeling human disease with pluripotent stem cells: from genome association to function.利用多能干细胞进行人类疾病建模:从基因组关联到功能。
Cell Stem Cell. 2013 Jun 6;12(6):656-68. doi: 10.1016/j.stem.2013.05.016.
3
A small molecule screen in stem-cell-derived motor neurons identifies a kinase inhibitor as a candidate therapeutic for ALS.在干细胞衍生的运动神经元中进行的小分子筛选确定了一种激酶抑制剂作为 ALS 的候选治疗药物。
Cell Stem Cell. 2013 Jun 6;12(6):713-26. doi: 10.1016/j.stem.2013.04.003. Epub 2013 Apr 18.
4
Enhanced efficiency of human pluripotent stem cell genome editing through replacing TALENs with CRISPRs.通过用CRISPRs替代TALENs提高人类多能干细胞基因组编辑效率。
Cell Stem Cell. 2013 Apr 4;12(4):393-4. doi: 10.1016/j.stem.2013.03.006.
5
Human embryonic stem cells differentiate into functional renal proximal tubular-like cells.人类胚胎干细胞分化为具有功能的肾近端管状样细胞。
Kidney Int. 2013 Apr;83(4):593-603. doi: 10.1038/ki.2012.442. Epub 2013 Feb 6.
6
Derivation of human induced pluripotent stem cells from patients with maturity onset diabetes of the young.从年轻起发病的成年型糖尿病患者中诱导多能干细胞的衍生。
J Biol Chem. 2013 Feb 22;288(8):5353-6. doi: 10.1074/jbc.C112.428979. Epub 2013 Jan 10.
7
A TALEN genome-editing system for generating human stem cell-based disease models.利用 TALEN 基因组编辑系统生成基于人类干细胞的疾病模型。
Cell Stem Cell. 2013 Feb 7;12(2):238-51. doi: 10.1016/j.stem.2012.11.011. Epub 2012 Dec 13.
8
Transgene-free disease-specific induced pluripotent stem cells from patients with type 1 and type 2 diabetes.1 型和 2 型糖尿病患者的无转基因疾病特异性诱导多能干细胞。
Stem Cells Transl Med. 2012 Jun;1(6):451-61. doi: 10.5966/sctm.2011-0044. Epub 2012 May 30.
9
Intrapatient variations in type 1 diabetes-specific iPS cell differentiation into insulin-producing cells.1 型糖尿病特异性诱导多能干细胞向胰岛素分泌细胞分化的患者内变异。
Mol Ther. 2013 Jan;21(1):228-39. doi: 10.1038/mt.2012.245. Epub 2012 Nov 27.
10
Induced pluripotent stem cells: the new patient?诱导多能干细胞:新的患者?
Nat Rev Mol Cell Biol. 2012 Nov;13(11):713-26. doi: 10.1038/nrm3448. Epub 2012 Oct 4.

新机遇:利用诱导多能性探索糖尿病和代谢疾病。

New opportunities: harnessing induced pluripotency for discovery in diabetes and metabolism.

机构信息

Section of Islet Cell and Regenerative Biology, Joslin Diabetes Center, Department of Medicine, Brigham and Women's Hospital, and Harvard Medical School, Boston, MA 02215, USA.

出版信息

Cell Metab. 2013 Dec 3;18(6):775-91. doi: 10.1016/j.cmet.2013.08.010. Epub 2013 Sep 12.

DOI:10.1016/j.cmet.2013.08.010
PMID:24035588
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3858409/
Abstract

The landmark discovery of induced pluripotent stem cells (iPSCs) by Shinya Yamanaka has transformed regenerative biology. Previously, insights into the pathogenesis of chronic human diseases have been hindered by the inaccessibility of patient samples. However, scientists are now able to convert patient fibroblasts into iPSCs and differentiate them into disease-relevant cell types. This ability opens new avenues for investigating disease pathogenesis and designing novel treatments. In this review, we highlight the uses of human iPSCs to uncover the underlying causes and pathological consequences of diabetes and metabolic syndromes, multifactorial diseases whose etiologies have been difficult to unravel using traditional methodologies.

摘要

山中伸弥(Shinya Yamanaka)发现诱导多能干细胞(iPSCs)这一具有里程碑意义的发现改变了再生生物学。在此之前,由于无法获得患者样本,慢性人类疾病发病机制的相关研究受到阻碍。然而,科学家现在能够将患者成纤维细胞转化为 iPSCs,并将其分化为与疾病相关的细胞类型。这种能力为研究疾病发病机制和设计新型治疗方法开辟了新途径。在这篇综述中,我们强调了使用人类 iPSCs 来揭示糖尿病和代谢综合征等多因素疾病的潜在病因和病理后果,这些疾病的病因用传统方法很难阐明。