Unravelling Endogenous MicroRNA System Dysfunction as a New Pathophysiological Mechanism in Machado-Joseph Disease

Machado-Joseph disease (MJD) is a genetic neurodegenerative disease caused by an expanded polyglutamine tract within the protein ataxin-3 (ATXN3). Despite current efforts, MJD’s mechanism of pathogenesis remains unclear and no disease-modifying treatment is available. Therefore, in this study, we in...

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Pubblicato in:Mol Ther
Autori principali: Carmona, Vitor, Cunha-Santos, Janete, Onofre, Isabel, Simões, Ana Teresa, Vijayakumar, Udaya, Davidson, Beverly L., Pereira de Almeida, Luís
Natura: Artigo
Lingua:Inglês
Pubblicazione: American Society of Gene & Cell Therapy 2017
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5383579/
https://ncbi.nlm.nih.gov/pubmed/28236575
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.01.021
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