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Gene-targeting pharmaceuticals for single-gene disorders

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 pharmaceuti...

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Bibliografiske detaljer
Udgivet i:Hum Mol Genet
Main Authors: Beaudet, Arthur L., Meng, Linyan
Format: Artigo
Sprog:Inglês
Udgivet: Oxford University Press 2016
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4802373/
https://ncbi.nlm.nih.gov/pubmed/26628634
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv476
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