Research article: “Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3”

Research article: “Toward allele-specific targeting therapy and pharmacodynamic marker for
spinocerebellar ataxia type 3”

Authors: 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, Sorina Gorcenco,
Andreas Puschmann, João Lemos, Cristina Januário, Mark S. LeDoux, Joseph H. Friedman,
James Polke, Robin Labrum, Vikram Shakkottai, Hayley S. McLoughlin, Henry L. Paulson, Takuya Konno, Osamu Onodera, Takeshi Ikeuchi, Mari Tada, Akiyoshi Kakita, John D. Fryer, Christin Karremo, Inês Gomes, John N. Caviness, Mark R. Pittelkow, Jan Aasly, Ronald F. Pfeiffer, Venka Veerappan, Eric R. Eggenberger, William D. Freeman, Josephine F. Huang, Ryan J. Uitti, Klaas J. Wierenga, Iris V. Marin Collazo, Philip W. Tipton, Jay A. van Gerpen, Marka van Blitterswijk, Guojun Bu, Zbigniew K. Wszolek, Paola Giunti, Leonard Petrucelli

Published: Science Translational Medicine (October 2020)