Review
Nature Reviews Genetics 7, 249-260 (April 2006) | doi:10.1038/nrg1828
There is a Correspondence and an Author Reply associated with this Article.
Focus on: Monogenic disorders
Human monogenic disorders — a source of novel drug targets
Ryan R. Brinkman1, Marie-Pierre Dubé2, Guy A. Rouleau3, Andrew C. Orr4 & Mark E. Samuels5 About the authors
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.
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Author affiliations
- British Columbia Cancer Research Centre, University of British Columbia, Vancouver, British Columbia V5Z 1C3, Canada.
- Montreal Heart Institute Research Centre, University of Montreal, Montreal, Quebec H1T 1C8, Canada.
- Department of Medicine, University of Montreal.
- Department of Ophthalmology & Visual Sciences, Dalhousie University, Halifax, Nova Scotia B3H 2Y9, Canada.
- Departments of Ophthalmology & Visual Sciences, Pediatrics and Psychiatry, Dalhousie University.
Correspondence to: Mark E. Samuels5 Email: Mark.Samuels@iwk.nshealth.ca
Published online 14 March 2006
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