siRNA Sequences & Methods for Treating Alzheimer Disease
Specific siRNA sequences and the addition of a 5' G have been demonstrated to modulate expression of Alzheimer disease-relevant mutant proteins, Tau & APP in vitro & in vivo.
UIRF Case #:04022
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Miller VM, Gouvion CM, Davidson BL, Paulson HL. Targeting Alzheimer's disease genes with RNA interference: an efficient strategy for silencing mutant alleles. Nucleic Acids Res. 2004 Jan 30;32(2):661-8.
Level of Development
General: in vitro
Researchers at the University of Iowa have identified a pair of siRNAs, one for the downregulation of mutant Tau (V337M) and the other for the downregulation of mutant amyloid precursor protein (swedish double mutation, K670N/M671L), which could serve as potential therapies in the treatment of Alzheimer disease. Several candidate siRNA sequences were developed that possessed the ability to inhibit expression of each of these mutant genes, and cell culture as well as mouse-based studies were used to identify the most effective sequence for inhibiting the expression of each gene. Studies describing the efficacy of these siRNA sequences also demonstrated the dose-dependent nature of their inhibitory activity. siRNA-mediated inhibition of mutant Tau and mutant APP could be used individually or in conjunction with each other in the clinical setting as needed to treat patients suffering from the effects of Alzheimer disease.
Inventor Web Site Link(s)