Technology Information

siRNA Sequences & Methods for Treating Huntington Disease

The use of specific sequences and a structural hairpin have been demonstrated to modulate expression of the Huntington's Disease protein in vitro & in vivo.
UIRF Case #:03054

Relevant PublicationsLevel of Development
Technology DescriptionInventor Web Site Link
Patent LinksContact Information
Other Information 

Relevant Publications

Harper SQ, et al. RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model. Proc Natl Acad Sci U S A. 2005 Apr 19; 102(16): 5820-5.

Level of Development

General: in vitro

Technology Description

Researchers at the University of Iowa have identified an siRNA-based method to potentially treat Huntington Disease. The invention consists of seven short sequences specific for Huntingtin mRNA and two others that have shown specificity for ataxin-1, which has been associated with spinocerebellar ataxia. These sequences have been shown to knock down expression of their target genes at the mRNA and protein levels both in vitro and in vivo. Furthermore, studies in mouse brain have clearly shown that treatment results in an elimination of the accumulation of Huntington's Disease protein inclusions in brain sections. Researchers have also demonstrated that the inclusion of a structural hairpin and overhang regions on the siRNA construct clearly increase their inhibitory activity.

Inventor Web Site Link(s)

Patent Link(s)

US8481710    US8524879    13/920,969    AU2005200827    AU2006210973    AU2009202278    CA2596588    SG109361    UK0502471.6    

Contact Information

Catherine Koh

6 Gilmore Hall, 112 N. Capitol Street, Iowa City, IA 52242     phone: 319-335-4546 fax: 319-335-4486 © University of Iowa 2007. All rights reserved.