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Limitations in a frataxin knockdown cell model for Friedreich ataxia in a high-throughput drug screen

BACKGROUND: Pharmacological high-throughput screening (HTS) represents a powerful strategy for drug discovery in genetic diseases, particularly when the full spectrum of pathological dysfunctions remains unclear, such as in Friedreich ataxia (FRDA). FRDA, the most common recessive ataxia, results fr...

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Autors principals: Calmels, Nadège, Seznec, Hervé, Villa, Pascal, Reutenauer, Laurence, Hibert, Marcel, Haiech, Jacques, Rustin, Pierre, Koenig, Michel, Puccio, Hélène
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2009
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC2744904/
https://ncbi.nlm.nih.gov/pubmed/19703283
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1471-2377-9-46
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