Epigenetic Therapy for Friedreich Ataxia

OBJECTIVE: To investigate whether a histone deacetylase inhibitor (HDACi) would be effective in an in vitro model for the neurodegenerative disease Friedreich ataxia (FRDA) and to evaluate safety and surrogate markers of efficacy in a phase I clinical trial in patients. METHODS: We used a human FRDA...

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Publicado en:Ann Neurol
Main Authors: Soragni, Elisabetta, Miao, Wenyan, Iudicello, Marco, Jacoby, David, De Mercanti, Stefania, Clerico, Marinella, Longo, Filomena, Piga, Antonio, Ku, Sherman, Campau, Erica, Du, Jintang, Penalver, Pablo, Rai, Myriam, Madara, Joseph C., Nazor, Kristopher, O’Connor, Melinda, Maximov, Anton, Loring, Jeanne F., Pandolfo, Massimo, Durelli, Luca, Gottesfeld, Joel M., Rusche, James R.
Formato: Artigo
Idioma:Inglês
Publicado: 2014
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC4361037/
https://ncbi.nlm.nih.gov/pubmed/25159818
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/ana.24260
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