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利用多能干细胞进行疾病建模:从实验室到临床,理解疾病。

Disease modeling using pluripotent stem cells: making sense of disease from bench to bedside.

机构信息

John F. Kennedy School of Government, 79 John F. Kennedy Street, Cambridge, MA 02138, USA.

出版信息

Swiss Med Wkly. 2011 Feb 9;141:w13144. doi: 10.4414/smw.2011.13144. eCollection 2011.

DOI:10.4414/smw.2011.13144
PMID:21308573
Abstract

New advances in human stem cell biology now permit the derivation of disease-specific induced pluripotent (iPS) stem cell lines, so-called "disease-in-a-dish" (DIAD) models. This is a promising approach for the study of disease phenotypes at the cellular and molecular level, both because such human cell lines may produce more faithful experimental models of disease than can be produced using non-human organisms, and because reprogrammed cell lines can provide a virtually infinite supply of cells without requiring additional tissue donation. However, expectations placed on this emerging technology privilege the laboratory over the clinic as the site for making sense of disease, thereby distracting from the socially embedded meanings of disease and reorienting how the goals of medicine are imagined. Here we identify and review the implications of this area of research for clinical approaches to disease. We argue that there is a central place for the larger medical community and patients in the very construction of experimental research programs and the expectations placed thereon. By attending to the constellation of social factors that inform understanding, treatments and experiences of disease, DIAD projects can be more effectively placed in the service of clinical goals, in both their research design and in the forms of innovation they claim to anticipate.

摘要

人类干细胞生物学的新进展现在允许衍生出疾病特异性诱导多能(iPS)干细胞系,即所谓的“疾病在培养皿中”(DIAD)模型。这是研究细胞和分子水平疾病表型的一种很有前途的方法,因为此类人类细胞系可能比使用非人类生物产生更真实的疾病实验模型,并且因为重编程细胞系可以在不要求额外组织捐献的情况下提供几乎无限量的细胞。然而,对这项新兴技术的期望将实验室置于临床之上,将实验室作为理解疾病的场所,从而分散了对疾病的社会嵌入意义的关注,并重新调整了对医学目标的想象。在这里,我们确定并审查了该研究领域对疾病临床方法的影响。我们认为,更大的医疗社区和患者在实验研究计划的构建及其期望中具有核心地位。通过关注告知疾病的理解、治疗和经验的一系列社会因素,DIAD 项目可以更有效地为临床目标服务,无论是在其研究设计还是在其声称预期的创新形式方面。

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引用本文的文献

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Proceedings: consideration of genetics in the design of induced pluripotent stem cell-based models of complex disease.会议记录:在基于诱导多能干细胞的复杂疾病模型设计中对遗传学的考量
Stem Cells Transl Med. 2014 Nov;3(11):1253-8. doi: 10.5966/sctm.2014-0191.
2
Specimen collection for induced pluripotent stem cell research: harmonizing the approach to informed consent.诱导多能干细胞研究的标本采集:协调知情同意书的方法。
Stem Cells Transl Med. 2012 May;1(5):409-21. doi: 10.5966/sctm.2012-0029. Epub 2012 May 8.
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Implications of aneuploidy for stem cell biology and brain therapeutics.
非整倍体对干细胞生物学和脑部治疗的影响。
Front Cell Neurosci. 2012 Sep 5;6:36. doi: 10.3389/fncel.2012.00036. eCollection 2012.
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An engineered approach to stem cell culture: automating the decision process for real-time adaptive subculture of stem cells.一种工程化的干细胞培养方法:实时自适应传代干细胞的决策过程自动化。
PLoS One. 2011;6(11):e27672. doi: 10.1371/journal.pone.0027672. Epub 2011 Nov 16.