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Using Genome Sequence to Enable the Design of Medicines and Chemical Probes
Chemical Reviews ( IF 62.1 ) Pub Date : 2018-01-11 00:00:00 , DOI: 10.1021/acs.chemrev.7b00504
Alicia J. Angelbello 1 , Jonathan L. Chen 1 , Jessica L. Childs-Disney 1 , Peiyuan Zhang 1 , Zi-Fu Wang 1 , Matthew D. Disney 1
Affiliation  

Rapid progress in genome sequencing technology has put us firmly into a postgenomic era. A key challenge in biomedical research is harnessing genome sequence to fulfill the promise of personalized medicine. This Review describes how genome sequencing has enabled the identification of disease-causing biomolecules and how these data have been converted into chemical probes of function, preclinical lead modalities, and ultimately U.S. Food and Drug Administration (FDA)-approved drugs. In particular, we focus on the use of oligonucleotide-based modalities to target disease-causing RNAs; small molecules that target DNA, RNA, or protein; the rational repurposing of known therapeutic modalities; and the advantages of pharmacogenetics. Lastly, we discuss the remaining challenges and opportunities in the direct utilization of genome sequence to enable design of medicines.

中文翻译:

使用基因组序列实现药物和化学探针的设计

基因组测序技术的飞速发展已使我们牢牢地步入了后基因组时代。生物医学研究的关键挑战是利用基因组序列来实现个性化医学的前景。这篇综述描述了基因组测序如何识别致病的生物分子,以及如何将这些数据转换为功能的化学探针,临床前先导模式以及最终获得美国食品药品监督管理局(FDA)批准的药物。特别是,我们专注于使用基于寡核苷酸的方式靶向致病性RNA。靶向DNA,RNA或蛋白质的小分子;合理地改变已知治疗方式;以及药物遗传学的优势。最后,
更新日期:2018-01-11
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