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Gene-targeting pharmaceuticals for single-gene disorders

30 Nov 2015

Abstract excerpt

The concept of orphan drugs for treatment of orphan genetic diseases is perceived enthusiastically at present, and this is leading to research investment on the part of governments, disease-specific foundations and industry. This review attempts to survey the potential to use traditional pharmaceuticals as opposed to biopharmaceuticals to treat single-gene disorders. The available strategies include the use of...

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PMID: 26628634
DOI: 10.1093/hmg/ddv476

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