• 主题
高级搜索  >
历史搜索 清空历史
首页> 外文期刊>EBioMedicine >2014: A Year of Breakthroughs for Translational Science
【24h】

2014: A Year of Breakthroughs for Translational Science

机译：2014年：转化科学突破的一年

获取原文
           
页面导航
  • 摘要
  • 著录项
  • 相似文献
  • 相关主题

摘要

2014 has been a remarkable year of scientific achievements, fromadvances in our understanding of molecular pathways of human diseasesto the development of new interventional tools to tackle them.Here, we highlight some promising initiatives and success stories fromthe past year with clear translational relevance. The list is by nomeans exhaustive but hopefully it provides a perspective on some ofthe achievements that are good examples of the types of researchEBioMedicine aims to foster.Although not an entirely new concept, a powerful new approachcalled CRISPR has been catapulted to center stage thisyear as a means of highly specific gene targeting and modification.Clustered regularly interspaced short palindromic repeats(CRISPR) are sequences naturally found in bacterial DNA thatwork with CRISPR-associated (Cas) nucleases and guide RNAsto protect the bacterial genomes from attacks by targeting specificsequences detected in invading bacteriophages. Proof-ofprincipleresearch has emerged recently, which harnesses thehigh sequence specificity of the system to cure genetic disordersin animal models as well as creating disease-free stemcells from affected patients. CRISPR also allows the creation ofmouse models with specific gene mutations for research purposeswithin much shorter timeframes than existing methods.Earlier this year, it was reported that the CRISPR-Cas systemhas successfully been applied as a tool for performing efficientand highly specific genome-wide screening in human cells,and vast improvements have been made in 2014 on increasingtarget specificity, which opens up limitless possibilities for interrogatinggene function relevant to human health anddiseases.
机译：2014年是非凡的科学成就，从对人类疾病分子途径的了解到发展新的干预手段来解决这些问题，在这里，我们着重介绍过去一年中一些有希望的举措和成功案例，这些案例与翻译具有明显的相关性。该清单虽然并不详尽，但希望能为EBioMedicine旨在促进的研究类型的一些成功实例提供一个观点。尽管这不是一个全新的概念，但一种名为CRISPR的功能强大的新方法已在今年成为了中心研究的热点。聚簇的规则间隔的短回文重复序列（CRISPR）是细菌DNA中天然存在的序列，可与CRISPR相关的（Cas）核酸酶协同工作，并指导RNA通过靶向侵袭噬菌体中检测到的特定序列来保护细菌基因组免受攻击。 。原理证明研究最近出现了，它利用了该系统的高序列特异性来治疗动物模型中的遗传性疾病，并从受影响的患者体内产生无病的干细胞。 CRISPR还可以在短于现有方法的时间范围内创建具有特定基因突变的小鼠模型用于研究目的。今年早些时候，有报道称CRISPR-Cas系统已成功地用作在全基因组中进行高效且高度特异性全基因组筛选的工具。人类细胞，2014年在提高靶标特异性方面取得了巨大进步，这为研究与人类健康和疾病相关的基因功能开辟了无限的可能性。

著录项

相似文献

  • 外文文献
  • 中文文献
  • 专利
获取原文

客服邮箱：kefu@zhangqiaokeyan.com

京公网安备：11010802029741号 ICP备案号：京ICP备15016152号-6 六维联合信息科技 (北京) 有限公司©版权所有

  • 客服微信

  • 服务号