Journal
DRUG DISCOVERY TODAY
Volume 23, Issue 2, Pages 382-394Publisher
ELSEVIER SCI LTD
DOI: 10.1016/j.drudis.2017.10.009
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Funding
- NATIONAL CENTER FOR ADVANCING TRANSLATIONAL SCIENCES [ZIATR000040] Funding Source: NIH RePORTER
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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.
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