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Highly specific ubiquitin-competing molecules effectively promote frataxin accumulation and partially rescue the aconitase defect in Friedreich ataxia cells

Friedreich ataxia is an inherited neurodegenerative disease that leads to progressive disability. There is currently no effective treatment and patients die prematurely. The underlying genetic defect leads to reduced expression of the mitochondrial protein frataxin. Frataxin insufficiency causes mit...

Ausführliche Beschreibung

Gespeichert in:
Hauptverfasser: Alessandra Rufini, Francesca Cavallo, Ivano Condò, Silvia Fortuni, Gabriella De Martino, Ottaviano Incani, Almerinda Di Venere, Monica Benini, Damiano Sergio Massaro, Gaetano Arcuri, Dario Serio, Florence Malisan, Roberto Testi
Format: Artigo
Sprache:Inglês
Veröffentlicht: Elsevier 2015-03-01
Schriftenreihe:Neurobiology of Disease
Schlagworte:
Friedreich ataxia
Frataxin
Ubiquitin
Orphan drug development
Online-Zugang:http://www.sciencedirect.com/science/article/pii/S0969996114003829
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