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One RNAi-Related Patent, Three Patent Applications Published by the USPTO


Title: Insect Control Agent. Number: 6,846,482. Filed: Aug. 13, 2001. Lead Inventor: Leo Liu, Cambria Biosciences.

The patent, its abstract states, covers “an expression vector engineered to produce double-stranded RNA within a pest to be controlled. The dsRNA inhibits expression of at least one gene within the pest, wherein inhibition of the gene exerts a deleterious effect upon the pest. For example, inhibition of the gene can lead to cessation of feeding, growth, or development and can cause death of the pest,” the abstract states.

“In a preferred embodiment of the invention the expression vector is a recombinant baculovirus that transcribes sense and antisense RNA under the control of the baculovirus IE-1 promoter and hr5 enhancer,” the abstract notes. “Preferred genes to be inhibited include essential genes, genes involved in neurotransmission, and genes that are targets for conventional pesticides. The invention discloses baculovirus transfer plasmids useful for producing the recombinant baculovirus. The invention further discloses methods and formulations involving the expression vector.”

Title: RNA Interference-Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid. Number: 20050020525. Filed: Jan. 14, 2004. Lead Inventor: James McSwiggen, Sirna Therapeutics.

The invention, the patent application’s abstract states, “concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid, short interfering RNA, double-stranded RNA, microRNA, and short-hairpin RNA molecules capable of mediating RNA interference against target nucleic acid sequences,” the abstract states.

“The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism,” the abstract adds.

Title: In Vivo Gene Silencing by Chemically Modified and Stable siRNA. Number: 20050020521. Filed: Sept. 25, 2003. Lead Inventor: Tariq Rana, University of Massachusetts Medical School (CytRx Laboratories).

According to the patent application’s abstract, the invention provides compositions for RNA interference and methods of use thereof. In particular, the invention provides small interfering RNAs having modification that enhance the stability of the siRNA without a concomitant loss in the ability of the siRNA to participate in RNA interference.”

The abstract notes that the invention also provides “siRNAs having modification that increase targeting efficiency. Modifications include chemical cross-linking between the two complementary strands of an siRNA and chemical modification of a 3’ terminus of a strand of an siRNA. Preferred modifications are internal modifications, for example, sugar modification, nucleobase modification, and/or backbone modifications,” it states. “Such modifications are also useful, [for example in improving] uptake of the siRNA by a cell. Functional and genomic and proteomic methods are featured, [as are] therapeutic methods.”

Title: Decreasing Gene Expression in a Mammalian Subject In Vivo Via AAV-Mediated RNAi Expression Cassette Transfer. Number: 20050019927. Filed: July 13, 2003. Lead Inventor: Markus Hildinger, University of Pennsylvania.

“The … invention relates to methods for decreasing gene expression by administering to a mammalian subject a recombinant adeno-associated viral vector in vivo with said vector comprising an RNA interference expression cassette whose RNA expression products directly or indirectly lead to a decrease in expression of the corresponding RNAi target gene,” the patent application’s abstract states. “Upon successful transduction with the recombinant adeno-associated viral vector, the RNA expression products of the RNAi expression cassette will decrease the cellular concentration of the mRNA transcripts of the RNAi target gene, thus resulting in decreased concentration of the protein encoded by the RNAi target gene.”

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