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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...

詳細記述

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書誌詳細
出版年:Hum Mol Genet
主要な著者: Beaudet, Arthur L., Meng, Linyan
フォーマット: Artigo
言語:Inglês
出版事項: Oxford University Press 2016
主題:
オンライン・アクセス: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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