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IP Update: Jul 15, 2010


Title: Control of Gene Expression

Patent Number: 7,754,697

Filed: Aug. 22, 2003

Lead Inventor: Michael Graham, Commonwealth Scientific and Industrial Research Organization (Benitec)

The invention, the patent's abstract states, "relates generally to a method of modifying gene expression and to synthetic genes for modifying endogenous gene expression in a cell, tissue, or organ of a transgenic organism, in particular a transgenic animal or plant. More particularly, the … invention utilizes recombinant DNA technology to post-transcriptionally modify or modulate the expression of a target gene in a cell tissue, organ, or whole organism, thereby producing novel phenotypes. Novel synthetic genes and genetic constructs which are capable of repressing, delaying, or otherwise reducing the expression of an endogenous gene or a target gene in an organism when introduced thereto are also provided."

Title: Therapeutic Targeting of Interleukins Using siRNA in Neutral Liposomes

Application Number: 20100172962

Filed: Feb. 29, 2008 PCT Filed: Feb. 29, 2008

Lead Inventor: William Merritt, University of Texas

The invention "relates to the fields of molecular biology and drug delivery," the patent application's abstract states. "In certain embodiments, the … invention provides compositions that include an siRNA targeted to an interleukin and a neutral lipid, and methods of treating a human subject with cancer involving administering to the subject a pharmaceutically effective amount of an interleukin-8 antagonist or a composition as set forth herein."

Title: Enhanced Biotherapeutic Production Using Inhibitory RNA

Application Number: 20100173359

Filed: July 11, 2008 PCT Filed: July 11, 2008

Lead Inventor: James Ladine, Thermo Fisher Scientific

The patent application, its abstract states, claims "compositions, kits, systems, equipment, and protocols [that] utilize synthetic siRNA [and have] a delivery-facilitating moiety in improved bioprocesses that enhance the production of biomaterials. The siRNA can target genes associated with … deleterious vector-derived genes; genes that confer non-optimal growth or differentiation properties to the cells; genes that can influence heterogeneity or post-translational modification pattern of the desirable gene product; genes that highly express non-desired proteins; genes that express proteins which interfere with purification of the desired protein; and other genes that can interfere with the bioprocess."

Title: Double-Stranded Nucleic Acid Molecule Cancer Cell Proliferation Inhibitor and Pharmaceutical Agent Suitable for Prevention or Treatment of Uterine Cancer, Breast Cancer, and Bladder Cancer

Application Number: 20100173405

Filed: Dec. 21, 2009

Lead Inventor: Satoshi Inoue, Saitama Medical University

The invention, the patent application's abstract states, comprises "a double-stranded nucleic acid molecule for suppressing the expression of at least one of COX7RP and Efp genes."

The dsRNA, the abstract states, "includes a sense strand [with] a nucleotide sequence corresponding to a target sequence indicated by [a predefined sequence, and] … an antisense strand which includes a nucleotide sequence complementary to that of the [specified] sense strand."

Titles: Extended Dicer-Substrate Agents and Methods for the Specific Inhibition of Gene Expression

Application Numbers: 20100173973, 20100173974

Filed: Dec. 18, 2009

Inventor: Bob Brown, Dicerna Pharmaceuticals

The inventions, the patent applications' abstracts state, comprise "compositions and methods for reducing expression of a target gene in a cell [by] contacting a cell with an isolated double-stranded nucleic acid in an amount effective to reduce expression of a target gene in a cell.

"The dsNAs of the invention possess a pattern of deoxyribonucleotides — in most embodiments, the pattern comprises at least one deoxyribonucleotide-deoxyribonucleotide base pair — designed to direct the site of Dicer enzyme cleavage within the dsNA molecule," it adds. "Deoxyribonucleotides of the dsNA molecules of the invention are located within a region of the dsNA that can be excised via Dicer cleavage to generate an active siRNA agent that no longer contains the deoxyribonucleotide pattern. Such DNA-extended Dicer-substrate siRNAs were demonstrated to be more effective RNA inhibitory agents than corresponding double-stranded RNA-extended DsiRNAs. DsiRNA agents were also found to tolerate guide-strand mismatches."

Title: RNA Interference-Mediated Inhibition of Stromal Cell-Derived Factor-1 Gene Expression Using Short Interfering Nucleic Acid

Application Number: 20100173976

Filed: March 16, 2010

Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)

The invention, the patent application's abstract states, "relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of stromal cell-derived factor-1 gene expression and/or activity."

The invention is also "directed to compounds, compositions, and methods relating to traits, diseases, and conditions that respond to the modulation of expression and/or activity of genes involved in SDF-1 gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases, and conditions," it adds. "Specifically, the invention relates to small nucleic acid molecules … capable of mediating or that mediate RNA interference against SDF-1 gene expression. Such small nucleic acid molecules are useful, for example, in providing compositions for treatment of traits, diseases, and conditions that can respond to modulation of SDF-1 expression in a subject, such as ocular disease, cancer and proliferative diseases, and any other disease, condition, trait, or indication that can respond to the level of SDF-1 in a cell or tissue."

Title: Tunable Genetic Switch for Regulating Gene Expression

Application Number: 20100175141

Filed: Oct. 19, 2007 PCT Filed: Oct. 19, 2007

Lead Inventor: James Collins, Boston University

The invention, the patent application's abstract states, relates to "methods, compositions, and systems for controlling the inducible expression of transgenes, while eliminating background expression of transgene expression. The … invention relates to methods of use of the compositions and systems as disclosed herein for controlling the inducible expression of transgenes while eliminating background expression of transgene expression, such as use in, for example, the generation of transgenic animals, use in [a] therapeutic application, and use in assays.

"In some embodiments, the … invention relates to a system of controlled expression of RNAi molecules which target binding sites in the untranslated regions of [a] transgene, thereby the expression of the transgene is modulated and leakiness is reduced," it notes. "The compositions and methods of the … invention can be used to for therapy, prophylaxis, research, and diagnostics in diseases and disorders which afflict mammalian species, generation of transgenic animals, in the study of biological processes, as well as for enhance performance of agricultural crops."

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