Inventors:
Richard Smith - Iowa City IA, US
Assignee:
University of Iowa Research Foundation - Iowa City IA
International Classification:
A61K 48/00, C07H 21/02, A61K 9/127, C12N 5/08, C12N 15/88
US Classification:
424450000, 514044000, 435325000, 435458000, 536023100, 435368000
Abstract:
This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of genes encoding proteins involved in deafness caused by dominant negative mechanism of action by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid, short interfering RNA, double-stranded RNA (dsRNA), micro-RNA (mRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of genes involved in deafness caused by dominant negative mechanism of action.