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Article

Finding chemical drugs for genetic diseases.

Drug discovery today - 1 Dec 2014

Sun Hui-Yong, Hou Ting-Jun, Zhang Hong-Yu

Abstract excerpt

Chemical drugs provide alternative treatments for genetic diseases in addition to gene therapy. Inherited diseases arising from gain-of-function (GOF) or loss-of-function (LOF) mutations of certain genes can be ameliorated by the antagonists and/or agonists of the target genes. However, a premise for this chemical therapeutic strategy is that the GOF/LOF mutations in drug targets have a negligible influence on...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 25256778
DOI: 10.1016/j.drudis.2014.09.013

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