Using Genome Sequence to Enable the Design of Medicines and Chemical Probes.
Using Genome Sequence to Enable the Design of Medicines and Chemical Probes.
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DOI：
10.1021/acs.chemrev.7b00504
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发表时间：
2018-02-28
期刊：
影响因子：
62.1
通讯作者：
Disney MD
中科院分区：
文献类型：
--
作者：
Angelbello AJ;Chen JL;Childs-Disney JL;Zhang P;Wang ZF;Disney MD
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.
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