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
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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
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)