Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3?

@article{Alves2010SilencingAM,
  title={Silencing ataxin-3 mitigates degeneration in a rat model of Machado-Joseph disease: no role for wild-type ataxin-3?},
  author={Sandro Alves and Isabel Nascimento-Ferreira and No{\"e}lle Dufour and Raymonde Hassig and Gwenna{\"e}lle Aur{\'e}gan and Cl{\'e}vio N{\'o}brega and Emmanuel Brouillet and Philippe Hantraye and Maria Conceiç{\~a}o Pedroso de Lima and Nicole D{\'e}glon and Lu{\'i}s Pereira de Almeida},
  journal={Human molecular genetics},
  year={2010},
  volume={19 12},
  pages={2380-94}
}
Machado-Joseph disease or spinocerebellar ataxia type 3 (MJD/SCA3) is a fatal, autosomal dominant disorder caused by a cytosine-adenine-guanine expansion in the coding region of the MJD1 gene. RNA interference has potential as a therapeutic approach but raises the issue of the role of wild-type ataxin-3 (WT ATX3) in MJD and of whether the expression of the wild-type protein must be maintained. To address this issue, we both overexpressed and silenced WT ATX3 in a rat model of MJD. We showed… CONTINUE READING

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