Emerging Opportunities for Target Discovery in Rare Cancers,Cell Chemical Biology

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Emerging Opportunities for Target Discovery in Rare Cancers
Cell Chemical Biology ( IF 6.6 ) Pub Date : 2017-09-21 , DOI: 10.1016/j.chembiol.2017.08.002
Tanaz Sharifnia ₁ , Andrew L Hong ₂ , Corrie A Painter ₁ , Jesse S Boehm ₁

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Rare cancers pose unique challenges to research due to their low incidence. Barriers include a scarcity of tissue and experimental models to enable basic research and insufficient patient accrual for clinical studies. Consequently, an understanding of the genetic and cellular features of many rare cancer types and their associated vulnerabilities has been lacking. However, new opportunities are emerging to facilitate discovery of therapeutic targets in rare cancers. Online platforms are allowing patients with rare cancers to organize on an unprecedented scale, tumor genome sequencing is now routinely performed in research and clinical settings, and the efficiency of patient-derived model generation has improved. New CRISPR/Cas9 and small-molecule libraries permit cancer dependency discovery in a rapid and systematic fashion. In parallel, large-scale studies of common cancers now provide reference datasets to help interpret rare cancer profiling data. Together, these advances motivate consideration of new research frameworks to accelerate rare cancer target discovery.

中文翻译：

罕见癌症靶标发现的新机遇

罕见癌症由于发病率低，给研究带来了独特的挑战。障碍包括缺乏进行基础研究的组织和实验模型以及临床研究的患者数量不足。因此，人们一直缺乏对许多罕见癌症类型的遗传和细胞特征及其相关脆弱性的了解。然而，新的机会正在出现，以促进罕见癌症治疗靶点的发现。在线平台使患有罕见癌症的患者能够以前所未有的规模组织起来，肿瘤基因组测序现在已在研究和临床环境中常规进行，并且源自患者的模型生成的效率也得到了提高。新的 CRISPR/Cas9 和小分子文库允许快速、系统地发现癌症依赖性。与此同时，对常见癌症的大规模研究现在提供了参考数据集，以帮助解释罕见的癌症分析数据。总之，这些进展促使人们考虑新的研究框架，以加速罕见癌症靶点的发现。

更新日期：2017-09-21

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