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Glimmers in illuminating the druggable genome

Nature Reviews Drug Discovery volume 17, pages 301–302 (2018)Cite this article

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Much biomedical research continues to focus on a small proportion of the human genome that has already been studied intensively. The Illuminating the Druggable Genome programme, initiated as a pilot project by the US National Institutes of Health Common Fund in 2014, is now being implemented to accelerate the investigation of subsets of understudied proteins that have potential therapeutic relevance.

Of the ~20,000 protein-coding genes in the human genome, few have been studied intensively. Furthermore, although ~3,000 of these genes have been estimated to be part of the 'druggable genome' — the subset of genes encoding proteins that have the ability to bind drug-like molecules — less than 700 are currently targeted by FDA-approved drugs1. This lack of attention to most of the proteome persists in the academic research community despite evidence that understudied proteins are often both interesting and important in biological processes2, and the pharmaceutical industry tends to focus on relatively well-understood proteins as targets for drug development in order to mitigate risk. Thus, there is a substantial unmet need to both expand basic knowledge and enlarge the cache of potential new drug targets through the functional characterization of understudied and poorly characterized members of druggable gene families.

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References

  1. Santos, R. et al. A comprehensive map of molecular drug targets. Nat. Rev. Drug Discov. 16, 19–34 (2017).

    CAS  Article  Google Scholar 

  2. Edwards, A. M. et al. Too many roads not taken. Nature 470, 163–165 (2011).

    CAS  Article  Google Scholar 

  3. Nguyen, D. T. et al. Collating protein information to shed light on the druggable genome. Nucleic Acids Res. 45, D995–D1002 (2017).

    CAS  Article  Google Scholar 

  4. Kroeze, W. K. et al. PRESTO-Tango as an open-source resource for interrogation of the druggable human GPCRome. Nat. Struct. Mol. Biol. 22, 362–369 (2015).

    CAS  Article  Google Scholar 

  5. Bruni, G. et al. Zebrafish behavioral profiling identifies multi-target antipsychotic-like compounds. Nat. Chem. Biol. 12, 559–566 (2016).

    CAS  Article  Google Scholar 

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Authors and Affiliations

  1. Office of the Director, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, 20892, Maryland, USA

    Griffin Rodgers

  2. Office of the Director, National Center for Advancing Translational Sciences, National Institutes of Health, Bethesda, 20817, Maryland, USA

    Christopher Austin & Christine Colvis

  3. Division of Program Coordination, Planning, and Strategic Initiatives, National Institutes of Health, Bethesda, 20892, Maryland, USA

    James Anderson

  4. Division of Diabetes, Endocrinology, and Metabolic Diseases. National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, 20892, Maryland, USA

    Aaron Pawlyk, Ronald Margolis & Jenna Baker

Authors
  1. Griffin Rodgers
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  2. Christopher Austin
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  3. James Anderson
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  4. Aaron Pawlyk
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  5. Christine Colvis
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  6. Ronald Margolis
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  7. Jenna Baker
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Corresponding author

Correspondence to Aaron Pawlyk.

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The authors declare no competing financial interests.

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FURTHER INFORMATION

GEN

Illuminating the Druggable Genome programme

NIH Common Fund

Pharos

Unexplored opportunities in the druggable human genome

Illuminating the Druggable Genome programme homepage

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Cite this article

Rodgers, G., Austin, C., Anderson, J. et al. Glimmers in illuminating the druggable genome. Nat Rev Drug Discov 17, 301–302 (2018). https://doi.org/10.1038/nrd.2017.252

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