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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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Detalhes bibliográficos
Principais autores: Calmels, Nadège, Seznec, Hervé, Villa, Pascal, Reutenauer, Laurence, Hibert, Marcel, Haiech, Jacques, Rustin, Pierre, Koenig, Michel, Puccio, Hélène
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2009
Assuntos:
Acesso em linha: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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