- Carlsbad CA, US - Austin TX, US Jing Liu - Austin TX, US Dongbo Yu - Austin TX, US David Corey - Dallas TX, US Eric E. Swayze - Encinitas CA, US
Assignee:
Ionis Pharmaceuticals, Inc. - Carlsbad CA Board of Regents of the University of Texas System - Austin TX
International Classification:
C07H 21/02
Abstract:
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.