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Aasly, Jan

0000-0002-0558-7686

Mayo Clinic

Norwegian University of Science and Technology, 7006 Trondheim, Norway.

Citations 1

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 .