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用于转化精准医学的临床前研究流程——来自一个跨学科脑肿瘤干细胞项目的经验

A Preclinical Pipeline for Translational Precision Medicine-Experiences from a Transdisciplinary Brain Tumor Stem Cell Project.

作者信息

Vargas-Toscano Andres, Janiak Christoph, Sabel Michael, Kahlert Ulf Dietrich

机构信息

Department of Neurosurgery, Medical Faculty, Heinrich-Heine University Düsseldorf, 40225 Düsseldorf, Germany.

Institut für Anorganische Chemie und Strukturchemie, Heinrich-Heine-University Düsseldorf, 40204 Düsseldorf, Germany.

出版信息

J Pers Med. 2021 Sep 7;11(9):892. doi: 10.3390/jpm11090892.

DOI:10.3390/jpm11090892
PMID:34575669
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC8472761/
Abstract

Efficient transdisciplinary cooperation promotes the rapid discovery and clinical application of new technologies, especially in the competitive sector of oncology. In this review, written from a clinical-scientist point of view, we used glioblastoma-the most common and most aggressive primary brain tumor as a model disease with a largely unmet clinical need, despite decades of intensive research-to promote transdisciplinary medicine. Glioblastoma stem-like cells (GSCs), a special tumoral cell population analogue to healthy stem cells, are considered largely responsible for the progression of the disease and the mediation of therapy resistance. The presented work followed the concept of translational science, which generates the theoretical backbones of translational research projects, and aimed to close the preclinical gap between basic research and clinical application. Thus, this generated an integrated translational precision medicine pipeline model based on recent theoretical and experimental publications, which supports the accelerated discovery and development of new paths in the treatment of GSCs. The work may be of interest to the general field of precision medicine beyond the field of neuro-oncology such as in Cancer Neuroscience.

摘要

高效的跨学科合作促进新技术的快速发现和临床应用,尤其是在竞争激烈的肿瘤学领域。在本综述中,我们从临床科学家的角度出发,以胶质母细胞瘤(最常见且最具侵袭性的原发性脑肿瘤)作为模型疾病,尽管经过数十年的深入研究,其临床需求仍 largely未得到满足,以此来推动跨学科医学。胶质母细胞瘤干细胞(GSCs)是一种类似于健康干细胞的特殊肿瘤细胞群体,被认为在很大程度上导致了疾病的进展并介导了治疗抗性。本研究遵循转化科学的概念,该概念为转化研究项目奠定理论基础,旨在弥合基础研究与临床应用之间的临床前差距。因此,基于近期的理论和实验出版物,生成了一个综合的转化精准医学管道模型,该模型支持加速发现和开发治疗GSCs的新途径。这项工作可能会引起神经肿瘤学领域之外的精准医学一般领域(如癌症神经科学)的兴趣。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f8ee/8472761/00ec7c0fd7d5/jpm-11-00892-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f8ee/8472761/00ec7c0fd7d5/jpm-11-00892-g001.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f8ee/8472761/00ec7c0fd7d5/jpm-11-00892-g001.jpg

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Developing and validating model systems for immuno-oncology.开发和验证免疫肿瘤学的模型系统。
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A first-in-human phase 0 clinical study of RNA interference-based spherical nucleic acids in patients with recurrent glioblastoma.
在复发性胶质母细胞瘤患者中进行基于 RNA 干扰的球形核酸的首次人体 0 期临床研究。
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Chinese Glioma Genome Atlas (CGGA): A Comprehensive Resource with Functional Genomic Data from Chinese Glioma Patients.中国脑胶质瘤基因组图谱(CGGA):来自中国脑胶质瘤患者的功能基因组数据的综合资源。
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