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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 ORCID, Hector Garcia-Moreno ORCID, Karen R. Jansen-West ORCID, Rana Hanna Al-Shaikh ORCID, Tania F. Gendron ORCID, Michael G. Heckman, Matthew R. Spiegel, Yari Carlomagno ORCID, Lillian M. Daughrity ORCID, Yuping Song, Judith A. Dunmore ORCID, Natalie Byron, Björn Oskarsson ORCID, Katharine A. Nicholson, Nathan P. Staff ORCID and other authors.
This paper was not found in any repository, but could be made available legally by the author.
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 .