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基于细胞的治疗方法:医学的下一个支柱。

Cell-based therapeutics: the next pillar of medicine.

机构信息

UCSF Center for Systems and Synthetic Biology, University of California, San Francisco, San Francisco, CA 94158, USA. fischbach@f schbach-group.org

出版信息

Sci Transl Med. 2013 Apr 3;5(179):179ps7. doi: 10.1126/scitranslmed.3005568.

DOI:10.1126/scitranslmed.3005568
PMID:23552369
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3772767/
Abstract

Two decades ago, the pharmaceutical industry-long dominated by small-molecule drugs-was revolutionized by the the advent of biologics. Today, biomedicine sits on the cusp of a new revolution: the use of microbial and human cells as versatile therapeutic engines. Here, we discuss the promise of this "third pillar" of therapeutics in the context of current scientific, regulatory, economic, and perceptual challenges. History suggests that the advent of cellular medicines will require the development of a foundational cellular engineering science that provides a systematic framework for safely and predictably altering and regulating cellular behaviors.

摘要

二十年前,制药行业长期以来一直由小分子药物主导,随着生物技术的出现而发生了革命性变化。如今,生物医学正处于新的革命的边缘:利用微生物和人体细胞作为多功能治疗引擎。在这里,我们在当前科学、监管、经济和观念挑战的背景下讨论了这种治疗方法的“第三大支柱”的前景。历史表明,细胞药物的出现将需要发展基础细胞工程科学,为安全、可预测地改变和调节细胞行为提供系统框架。

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Microbiome. 2013 Jan 9;1(1):3. doi: 10.1186/2049-2618-1-3.
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Duodenal infusion of donor feces for recurrent Clostridium difficile.经十二指肠输注供体粪便治疗复发性艰难梭菌感染。
N Engl J Med. 2013 Jan 31;368(5):407-15. doi: 10.1056/NEJMoa1205037. Epub 2013 Jan 16.
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RNA-guided human genome engineering via Cas9.通过 Cas9 进行 RNA 引导的人类基因组工程。
Science. 2013 Feb 15;339(6121):823-6. doi: 10.1126/science.1232033. Epub 2013 Jan 3.
4
Human neural stem cells induce functional myelination in mice with severe dysmyelination.人类神经干细胞可诱导严重脱髓鞘小鼠产生功能性髓鞘形成。
Sci Transl Med. 2012 Oct 10;4(155):155ra136. doi: 10.1126/scitranslmed.3004371.
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FDA oversight of cell therapy clinical trials.FDA 对细胞治疗临床试验的监管。
Sci Transl Med. 2012 Aug 29;4(149):149fs31. doi: 10.1126/scitranslmed.3004131.
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A decade of cell therapy clinical trials (2000-2010).细胞疗法临床试验的十年(2000 - 2010年)。
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