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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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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Beaudet, Arthur L., Meng, Linyan
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2016
Assuntos:
Acesso em linha: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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