Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3
Science Translational Medicine | October 21, 2020 | Volume: 12 | Issue: 566 | Pages: No page available
10.1126/scitranslmed.abb7086
| PMID 33087504
PolyQ ATXN3 is a pharmacodynamic marker for SCA3, and a SNP associated with the expanded allele may serve to specifically target mutant ATXN3 .