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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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Detalles Bibliográficos
Autores principales: Calmels, Nadège, Seznec, Hervé, Villa, Pascal, Reutenauer, Laurence, Hibert, Marcel, Haiech, Jacques, Rustin, Pierre, Koenig, Michel, Puccio, Hélène
Formato: Texto
Lenguaje:English
Publicado: BioMed Central 2009
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2744904/
https://www.ncbi.nlm.nih.gov/pubmed/19703283
http://dx.doi.org/10.1186/1471-2377-9-46