Rational drug repurposing using sscMap analysis in a HOX-TALE model of leukemia

Drug discovery and development are often hampered by lack of target identification and clinical tractability. Repurposing of approved drugs to life-threatening diseases such as leukemia is emerging as a promising alternative approach. Connectivity mapping systems link approved drugs with disease-rel...

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Bibliographic Details
Main Authors: Kettyle, Laura M.J., Liberante, Fabio G., Thompson, Alexander
Format: Article
Published: Springer Verlag 2014
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Online Access:https://eprints.nottingham.ac.uk/42819/
Description
Summary:Drug discovery and development are often hampered by lack of target identification and clinical tractability. Repurposing of approved drugs to life-threatening diseases such as leukemia is emerging as a promising alternative approach. Connectivity mapping systems link approved drugs with disease-related gene signatures. Relevant preclinical models provide essential tools for system validation and proof-of-concept studies. Herein we describe procedures aimed at generating disease-based gene signatures and applying them to established cross-referencing databases of potential candidate drugs. As a proof of principle, we present the identification of Entinostat as a candidate drug for the treatment of HOX TALE-related leukemia.