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Five RNAi-Related Patent Applications Published by the US Patent Office: Sep 24, 2004

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Title: SiRNA Research Tool Kit. Number: 20040181821. Filed: Feb. 27, 2004. Lead Inventor: Jiadong Zhou.

The patent application, its abstract states, covers “a kit … for gene functional studies, which allows users to produce siRNA via siRNA expression cassettes; efficiently introduce the siRNA expression cassette into cultured mammalian cells; evaluate the transfection efficiency; and evaluate the siRNA synthetic efficiency.

“The kit includes a siRNA synthetic system; a transfection reagent; a PCR primer with a specific fluorescent dye tag for tracking down the siRNA delivery pathway; and a reporter gene cassette such as GFP gene expression cassette for easily selecting the cells that are successfully transfected with the siRNA in the whole cell pool,” the abstract adds.


Title: Compositions and Methods for siRNA Inhibition of HIF-1 Alpha. Number: 20040180357. Filed: Oct. 31, 2003. Lead Inventor: Samuel Jotham Reich, University of Pennsylvania (Acuity Pharmaceuticals).

The patent application covers “RNA interference using small interfering RNAs which target HIF-1 alpha mRNA inhibit expression of the HIF-1 alpha gene,” its abstract states. “As HIF-1 alpha is a transcriptional regulator of VEGF, expression of VEGF is also inhibited. Control of VEGF production through siRNA-mediated down-regulation of HIF-1 alpha can be used to inhibit angiogenesis, in particular in diseases such as diabetic retinopathy, age related macular degeneration, and ... cancer.”


Title: Interfering RNA Molecules. Number: 20040180351. Filed: Oct. 31, 2003. Lead Inventor: Klaus Giese, Atugen.

“The present invention is related to a ribonucleic acid comprising a double stranded structure,” the patent application’s abstract states.

The first strand of this structure comprises a “stretch of contiguous nucleotides … [which] is at least partially complementary to a target nucleic acid,” the abstract notes. The second strand “comprises … a stretch of contiguous nucleotides [that is] at least partially identical to a target nucleic acid.” The abstract adds that the “double stranded structure is blunt ended.”


Title: Methods and Compositions for Silencing Genes Without Inducing Toxicity. Number: 20040180438. Filed: April 28, 2003. Lead Inventor: Catherine Pachuk, Nucleonics.

According to the patent application’s abstract, the invention comprises “methods of post-transcriptional gene silencing which involve the use of a first dsRNA having substantial sequence identity to a target nucleic acid and a short, second dsRNA, which inhibits dsRNA-mediated toxicity. These methods can be used to prevent or treat a disease or infection by silencing a gene associated with the disease or infection,” the abstract notes.

The invention also provides “methods for identifying nucleic acid sequences that modulate a detectable phenotype, including the function of a cell, the expression of a gene, or the biological activity of a target polypeptide,” the abstract states.


Title: Methods of Assaying for Cell Cycle Modulators Using Components of the Ubiquitin Ligation Cascade. Number: 20040180353. Filed: Aug. 28, 2003. Lead Inventor: Robert Booher, Rigel Pharmaceuticals.

“The present invention relates to regulation of the cell cycle,” the patent application’s abstract states. “More particularly, the present invention is directed to nucleic acids encoding components of the ubiquitin ligation pathway, e.g., ubiquitin and ubiquitin-like molecules, E1, E2, and E3 proteins and their substrates, which are involved in modulation of cell cycle arrest.”

Also covered by the application are “methods for identifying and using agents, including small molecule chemical compositions, antibodies, peptides, cyclic peptides, nucleic acids, RNAi, antisense nucleic acids, and ribozymes, that modulate cell cycle arrest via modulation of the ubiquitin ligation pathway; as well as to the use of expression profiles and compositions in diagnosis and therapy related to cell cycle regulation and modulation of cellular proliferation, e.g., for treatment of cancer and other diseases of cellular proliferation,” the abstract adds.

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