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Pathway and network-based strategies to translate genetic discoveries into effective therapies

View ORCID ProfileCasey S. Greene, Benjamin F. Voight
doi: https://doi.org/10.1101/051524
Casey S. Greene
1Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of Pennsylvania
3Institute for Translational Medicine and Therapeutics, Perelman School of Medicine, University of Pennsylvania.
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  • For correspondence: csgreene@upenn.edu
Benjamin F. Voight
1Department of Systems Pharmacology and Translational Therapeutics, Perelman School of Medicine, University of Pennsylvania
2Department of Genetics, Perelman School of Medicine, University of Pennsylvania
3Institute for Translational Medicine and Therapeutics, Perelman School of Medicine, University of Pennsylvania.
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ABSTRACT

One way to design a drug is to attempt to phenocopy a genetic variant that is known to have the desired effect. In general, drugs that are supported by genetic associations progress further in the development pipeline. However, the number of associations that are candidates for development into drugs is limited because many associations are in noncoding regions or difficult to target genes. Approaches that overlay information from pathway databases or biological networks can expand the potential target list. In cases where the initial variant is not targetable or there is no variant with the desired effect, this may reveal new means to target a disease. In this review we discuss recent examples in the domain of pathway and network-based drug repositioning from genetic associations. We highlight important caveats and challenges for the field, and we discuss opportunities for further development.

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The copyright holder for this preprint is the author/funder, who has granted bioRxiv a license to display the preprint in perpetuity. All rights reserved. No reuse allowed without permission.
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Posted May 03, 2016.
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Pathway and network-based strategies to translate genetic discoveries into effective therapies
Casey S. Greene, Benjamin F. Voight
bioRxiv 051524; doi: https://doi.org/10.1101/051524
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Pathway and network-based strategies to translate genetic discoveries into effective therapies
Casey S. Greene, Benjamin F. Voight
bioRxiv 051524; doi: https://doi.org/10.1101/051524

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