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Drug Discov Today. 2018 Feb;23(2):382-394. doi: 10.1016/j.drudis.2017.10.009. Epub 2017 Oct 18.

Computational drug repositioning for rare diseases in the era of precision medicine.

Author information

1
National Center for Toxicological Research, US Food and Drug Administration, Jefferson, AR 72079, USA; University of Arkansas at Little Rock, Little Rock, AR 72204, USA.
2
ApconiX, BioHub at Alderley Park, Alderley Edge SK10 4TG, UK; University of Birmingham, Edgbaston, Birmingham B15 2TT, UK.
3
National Center for Advancing Translational Sciences, National Institutes of Health Rockville, MD 20850, USA.
4
SAS Institute Inc., Cary, NC, USA.
5
National Center for Toxicological Research, US Food and Drug Administration, Jefferson, AR 72079, USA. Electronic address: weida.tong@fda.hhs.gov.
6
National Center for Toxicological Research, US Food and Drug Administration, Jefferson, AR 72079, USA. Electronic address: zhichao.liu@fda.hhs.gov.

Abstract

There are tremendous unmet needs in drug development for rare diseases. Computational drug repositioning is a promising approach and has been successfully applied to the development of treatments for diseases. However, how to utilize this knowledge and effectively conduct and implement computational drug repositioning approaches for rare disease therapies is still an open issue. Here, we focus on the means of utilizing accumulated genomic data for accelerating and facilitating drug repositioning for rare diseases. First, we summarize the current genome landscape of rare diseases. Second, we propose several promising bioinformatics approaches and pipelines for computational drug repositioning for rare diseases. Finally, we discuss recent regulatory incentives and other enablers in rare disease drug development and outline the remaining challenges.

PMID:
29055182
DOI:
10.1016/j.drudis.2017.10.009
[Indexed for MEDLINE]

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