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Drug repositioning can accelerate discovery of pharmacological chaperones

A promising strategy for the treatment of genetic diseases, pharmacological chaperone therapy, has been proposed recently. It exploits small molecules which can be administered orally, reach difficult tissues such as the brain and have low cost. This strategy has a vast field of application. In orde...

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Bibliografiska uppgifter
I publikationen:Orphanet J Rare Dis
Huvudupphovsmän: Hay Mele, Bruno, Citro, Valentina, Andreotti, Giuseppina, Cubellis, Maria Vittoria
Materialtyp: Artigo
Språk:Inglês
Publicerad: BioMed Central 2015
Ämnen:
Länkar:https://ncbi.nlm.nih.gov/pmc/articles/PMC4429356/
https://ncbi.nlm.nih.gov/pubmed/25947946
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-015-0273-2
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