Human monogenic disorders - A source of novel drug targets

Ryan R. Brinkman, Marie Pierre Dubé, Guy A. Rouleau, Andrew C. Orr, Mark E. Samuels

Research output: Contribution to journalReview articlepeer-review

78 Citations (Scopus)

Abstract

The decrease in new drug applications and approvals over the past several years results from an underlying crisis in drug target identification and validation. Model organisms are being used to address this problem, in combination with novel approaches such as the International HapMap Project. What has been underappreciated is that discovery of new drug targets can also be revived by traditional Mendelian genetics. A large fraction of the human gene repertoire remains phenotypically uncharacterized, and is likely to encode many unanticipated and novel phenotypes that will be of interest to pharmaceutical and biotechnological drug developers.

Original languageEnglish
Pages (from-to)249-260
Number of pages12
JournalNature Reviews Genetics
Volume7
Issue number4
DOIs
Publication statusPublished - Apr 2006

Bibliographical note

Funding Information:
Thanks to V. McKusick and J. Amberger for data from the Online Mendelian Inheritance in Man. Thanks to M. Ludman, S. Dyack and D. Skidmore for valuable discussions of clinical genetics phenotypes. Thanks to P. Goldberg, R. Sherrington, S. Pimstone for discussions of therapeutic targets. Thanks to S. Chanda for logistic assistance. R.R.B. is supported by a Michael Smith Foundation for Health Research (MSFHR) Research Unit Infrastructure award. M.E.S. is supported by the IWK Health Centre, Capital District Health Authority and Dalhousie University Faculty of Medicine.

ASJC Scopus Subject Areas

  • Molecular Biology
  • Genetics
  • Genetics(clinical)

Fingerprint

Dive into the research topics of 'Human monogenic disorders - A source of novel drug targets'. Together they form a unique fingerprint.

Cite this