Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy.

@article{Keiser2015BroadDO,
  title={Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy.},
  author={Megan S. Keiser and Jeffrey H. Kordower and Pedro Gonzalez-Alegre and Beverly L. Davidson},
  journal={Brain : a journal of neurology},
  year={2015},
  volume={138 Pt 12},
  pages={3555-66}
}
Spinocerebellar ataxia type 1 is one of nine polyglutamine expansion diseases and is characterized by cerebellar ataxia and neuronal degeneration in the cerebellum and brainstem. Currently, there are no effective therapies for this disease. Previously, we have shown that RNA interference mediated silencing of ATXN1 mRNA provides therapeutic benefit in mouse models of the disease. Adeno-associated viral delivery of an engineered microRNA targeting ATXN1 to the cerebella of well-established mouse… CONTINUE READING
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