editiorial commentary genomics and drug discovery the next frontier in precision medicine:(editiorial评论在精密医学基因组学和药物发现的下一个前沿).pdfVIP
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editiorial commentary genomics and drug discovery the next frontier in precision medicine:(editiorial评论在精密医学基因组学和药物发现的下一个前沿)
T R E N D S I N C A R D I O V A S C U L A R M E D I C I N E 2 7 ( 2 0 1 7 ) 2 0 3 – 2 0 6
Available online at
/locate/tcm
Editiorial Commentary: Genomics and drug
discovery: The next frontier in precision medicine
Dan M. Roden, MD*
Departments of Medicine, Pharmacology, and Biomedical Informatics, Vanderbilt University Medical Center, 2215B Garland Ave,
1285 MRBIV, Nashville, TN 37232-0575
Precision medicine has come to mean many things to many critical role of HMG-CoA reductase in cholesterol synthesis
people—a perfectly precise diagnosis and treatment, incor- and the beneficial effects of HMG-CoA reductase inhibition
poration of electronic health records (EHRs), and genomic by statins on plasma LDL-C and ultimately on cardiovascular
information into routine clinical care, or expanded consid- morbidity and mortality. A second success story is the
eration of sociocultural factors in decision-making [1]. In development of PCSK9 inhibitors [6]: this narrative started
this issue of Trends in Cardiovascular Medicine, Stitziel and with the identification of “gain-of-function” mutations in
Kathiresan [2] outline an emerging frontier in this area, the PCSK9, encoding the proprotein convertase subtilisin/kexin
use of human genetics to identify and validate new targets type 9 serine protease, as causes of FH [7]. Hobbs et al. then
for drug development. This may turn out to be an especially reasoned, and subsequently went on to prove [8], that
interesting area since new drug development is extraordi- nonsense variants (such as
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