Published in
American Association for the Advancement of Science, Science Translational Medicine, 566(12), 2020
DOI: 10.1126/scitranslmed.abb7086
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Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3
Journal article published in 2020 by
Mercedes Prudencio 
,
Hector Garcia-Moreno ,
Karen R. Jansen-West ,
Rana Hanna Al-Shaikh ,
Tania F. Gendron ,
Michael G. Heckman,
Matthew R. Spiegel,
Yari Carlomagno ,
Lillian M. Daughrity ,
Yuping Song,
Judith A. Dunmore ,
Natalie Byron,
Björn Oskarsson ,
Katharine A. Nicholson,
Nathan P. Staff
and other authors.
,
Hector Garcia-Moreno ,
Karen R. Jansen-West ,
Rana Hanna Al-Shaikh ,
Tania F. Gendron ,
Michael G. Heckman,
Matthew R. Spiegel,
Yari Carlomagno ,
Lillian M. Daughrity ,
Yuping Song,
Judith A. Dunmore ,
Natalie Byron,
Björn Oskarsson ,
Katharine A. Nicholson,
Nathan P. Staff
and other authors.
This paper was not found in any repository, but could be made available legally by the author.
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Abstract
PolyQ ATXN3 is a pharmacodynamic marker for SCA3, and a SNP associated with the expanded allele may serve to specifically target mutant ATXN3 .