Citations 1
Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3
Prudencio, Mercedes , García‐Moreno, Héctor , Jansen‐West, Karen , Al‐Shaikh, Rana Hanna , Gendron, Tania F. , Spiegel, Matthew R. , Carlomagno, Yari , Daughrity, Lillian M. , Song, Yuping , Dunmore, Judith A. , Byron, Natalie, Oskarsson, Björn , Nicholson, Katharine, Staff, Nathan P. , Gorcenco, Sorina , Puschmann, Andreas , Lemos, João , Januário, Cristina , LeDoux, Mark S. , Polke, James, Labrum, Robin, McLoughlin, Hayley S. , Paulson, Henry L. , Onodera, Osamu , Tada, Mari , Kakita, Akiyoshi , Fryer, John Denis , Karremo, Christin, Gomes, Inês , Caviness, John N., Pittelkow, Mark R. , Aasly, Jan , Pfeiffer, Ronald F. , Veerappan, Venka , Eggenberger, Eric , Huang, Josephine F. , Uitti, Ryan J. , Wierenga, Klaas J. , Collazo, I. Vanessa Marin , Tipton, Philip W. , Gerpen, Jay A. van, Blitterswijk, Marka van , Bu, Guojun , Petrucelli, Leonard , Jansen-West, Karen R , Carlomagno, Yari , Daughrity, Lillian M , Dunmore, Judith A , Oskarsson, Björn , Staff, Nathan P , Gorcenco, Sorina , Puschmann, Andreas , Lemos, João , Januário, Cristina , Shakkottai, Vikram , McLoughlin, Hayley S , Paulson, Henry L , Konno, Takuya , Onodera, Osamu , Ikeuchi, T , Tada, Mari , Gomes, Inês , Pittelkow, Mark R , Aasly, Jan , Eggenberger, Eric R, Freeman, William D , Huang, Josephine F , Marin Collazo, Iris V , Tipton, Philip W , van Blitterswijk, Marka , Bu, Guojun , Wszolek, Z.K. , Giunti, Paola , Petrucelli, Leonard , Prudencio, Mercedes , Gendron, Tania F , Hanna Al-Shaikh, Rana , Heckman, Michael G. , Friedman, Joseph H. , Konno, Takuya , Ikeuchi, Takeshi , Freeman, William D. , Wszołek, Zbigniew K.
Science Translational Medicine | October 21, 2020 | Volume: 12 | Issue: 566 | Pages: No page available
PolyQ ATXN3 is a pharmacodynamic marker for SCA3, and a SNP associated with the expanded allele may serve to specifically target mutant ATXN3 .