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Using Genome Sequence to Enable the Design of Medicines and Chemical Probes.

ABSTRACT: 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.

SUBMITTER: Angelbello AJ

PROVIDER: S-EPMC5989578 | biostudies-literature | 2018 Feb

REPOSITORIES: biostudies-literature

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Using Genome Sequence to Enable the Design of Medicines and Chemical Probes.

Angelbello Alicia J AJ Chen Jonathan L JL Childs-Disney Jessica L JL Zhang Peiyuan P Wang Zi-Fu ZF Disney Matthew D MD

Chemical reviews 20180111 4

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 ...[more]

PMID: 29322778

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