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Three RNAi-Related Patent Applications Published by the US Patent Office

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Title: Screening Assays for Targets and Drugs Useful in Treatment and Prevention of Lipid Metabolism Disorders. Number: 20040216174. Filed: March 12, 2004. Lead Inventor: Siegfried Hekimi, McGill University.

“The invention relates to the use of C. elegans as a model for identifying genes that are involved in lipid or lipoprotein metabolism and which can serve as drug targets,” the patent application’s abstract states. “The invention also provides a system for screening drugs useful in the treatment and prevention of diseases associated with undesirable or abnormal levels of lipids or lipoproteins.”


Title: Allele-Targeted RNA Interference. Number: 20040214198. Filed: Nov. 17, 2003. Inventor: Tariq Rana, University of Massachusetts Medical School.

“The … invention provides siRNAs with modified bases in the antisense strand … and methods for using the modified siRNAs to selectively down-regulate the expression of a mutant allele, even when the mutant mRNA differs from wild-type by only a single nucleotide,” the patent application’s abstract states.


Title: Knockout Reagent Surrogate Screening Assay. Number: 20040214181. Filed: April 25, 2003. Inventor: Vic Evan Myer, Akceli.

The invention “features a method and array for high throughput analysis of candidate knockout reagents in order to identify those capable of gene silencing,” the patent application’s abstract states.

The application states that the method involves the following steps: “depositing a plurality of nucleic acid molecules onto a surface in discrete, defined locations, wherein at each location is deposited a plurality of first nucleic acid molecules, wherein said first nucleic acid molecules comprise candidate knockout reagents or encode candidate knockout reagents, and a plurality of second nucleic acid molecules, wherein each second nucleic acid molecule comprises: a promoter; a reporter gene comprising a 5’ or 3’ untranslated region, said reporter gene operably linked to said promoter for expression in said cell; and a target nucleic acid derived from said target gene, said target nucleic acid located within said untranslated region, wherein different first nucleic acid molecules are deposited at different discrete, defined locations.”

According to the application, the method then calls for “contacting [the] cells with said nucleic acid molecules under appropriate conditions for entry of the nucleic acid molecules into said cells, whereby said nucleic acid molecules are introduced into the cells in the location in which each of the nucleic acid molecules was deposited.”

Finally, the method requires “determining whether a first nucleic acid molecule at a discrete, defined location reduces expression of said reporter gene, relative to expression of said reporter gene in a cell in the absence of said first nucleic acid molecule, wherein reduction of expression of said reporter gene identifies said first nucleic acid molecule at said discrete, defined location as a nucleic acid molecule capable of gene silencing.”