Toward allele-specific targeting therapy and pharmacodynamic marker for spinocerebellar ataxia type 3
针对3型脊髓小脑性共济失调的等位基因特异性靶向治疗和药效学标志物研究
期刊:Science Translational Medicine
影响因子:14.6
doi:10.1126/scitranslmed.abb7086
Prudencio, Mercedes; Garcia-Moreno, Hector; Jansen-West, Karen R; Al-Shaikh, Rana Hanna; Gendron, Tania F; Heckman, Michael G; Spiegel, Matthew R; Carlomagno, Yari; Daughrity, Lillian M; Song, Yuping; Dunmore, Judith A; Byron, Natalie; Oskarsson, Björn; Nicholson, Katharine A; Staff, Nathan P; Gorcenco, Sorina; Puschmann, Andreas; Lemos, João; Januário, Cristina; LeDoux, Mark S; Friedman, Joseph H; Polke, James; Labrum, Robin; Shakkottai, Vikram; McLoughlin, Hayley S; Paulson, Henry L; Konno, Takuya; Onodera, Osamu; Ikeuchi, Takeshi; Tada, Mari; Kakita, Akiyoshi; Fryer, John D; Karremo, Christin; Gomes, Inês; Caviness, John N; Pittelkow, Mark R; Aasly, Jan; Pfeiffer, Ronald F; Veerappan, Venka; Eggenberger, Eric R; Freeman, William D; Huang, Josephine F; Uitti, Ryan J; Wierenga, Klaas J; Marin Collazo, Iris V; Tipton, Philip W; van Gerpen, Jay A; van Blitterswijk, Marka; Bu, Guojun; Wszolek, Zbigniew K; Giunti, Paola; Petrucelli, Leonard