METHODS AND COMPOUNDS USEFUL IN CONDITIONS RELATED TO REPEAT EXPANSION
摘要
Described are compounds and methods useful for the treatment and investigation of diseases and disorders associated with expanded repeat-containing RNA molecules. In certain embodiments, compounds and methods useful for the modulation of ATXN-3 pre-mRNA are described. In certain embodiments, compounds and methods useful for the modulation of ATN-1 mRNA are described.
申请公布号
WO2015017675(A2)
申请公布日期
2015.02.05
申请号
WO2014US49197
申请日期
2014.07.31
申请人
ISIS PHARMACEUTICALS, INC.;BOARD OF REGENTS OF THE UNIVERSITY OF TEXAS SYSTEM
发明人
PRAKASH, THAZHA, P.;HU, JIAXIN;LIU, JING;YU, DONGBO;COREY, DAVID;SWAYZE, ERIC, E.