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IP Update: May 27, 2010

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Title: Diagnosis and Treatment of Cancers with microRNA Located In or Near Cancer-Associated Chromosomal Features

Patent Number: 7,723,030

Filed: July 29, 2005

Lead Inventor: Carlo Croce, Thomas Jefferson University

According to the patent's abstract, "microRNA genes are highly associated with chromosomal features involved in the etiology of different cancers. The perturbations in the genomic structure or chromosomal architecture of a cell caused by these cancer-associated chromosomal features can affect the expression of the miR genes located in close proximity to that chromosomal feature. Evaluation of miR gene expression can therefore be used to indicate the presence of a cancer-causing chromosomal lesion in a subject.

"As the change in miR gene expression level caused by a cancer-associated chromosomal feature may also contribute to cancerigenesis, a given cancer can be treated by restoring the level of miR gene expression to normal," the abstract notes. Additionally, "microRNA expression profiling can be used to diagnose cancer and predict whether a particular cancer is associated with an adverse prognosis. The identification of specific mutations associated with genomic regions that harbor miR genes in CLL patients provides a means for diagnosing CLL and possibly other cancers."


Title: Methods and Compositions for Treating Pachyonychia Congenita

Patent Number: 7,723,314

Filed: Aug. 24, 2006

Lead Inventor: Roger Kaspar, TransDerm

The patent, its abstract states, claims "a method for treating pachyonychia congenita by identifying a genetic mutation contributing to PC, preparing a siRNA sequence that inhibits expression of the identified mutation, and administering the siRNA to a cell of a subject afflicted with PC. The siRNA treatment can include silencing of both the mutated and associated wild-type genes for a specific keratin. Specific mutations and inhibitory sequences are identified."


Title: Compositions and Methods of RNAi Therapeutics for Treatment of Cancer and Other Neovascularization Diseases

Patent Number: 7,723,316

Filed: June 29, 2007

Lead Inventor: Yijia Liu, Intradigm

The patent, its abstract states, claims "compositions and methods … for treatment of diseases involving unwanted neovascularization. The invention provides treatments that control [neovascularization] through selective inhibition of pro-angiogenic biochemical pathways, including inhibition of the VEGF pathway gene expression and inhibition localized at pathological [neovascularization] tissues. Tissue targeted nanoparticle compositions comprising polymer conjugates and nucleic acid molecules that induce RNA interference are provided.

"The nanoparticle compositions of the invention can be used alone or in combination with other therapeutic agents such as VEGF pathway antagonists," the abstract adds. "The compositions and methods can be used for the treatment of [neovascularization] diseases such as cancer, ocular disease, arthritis, and inflammatory diseases."


Title: RNAi Modulation of ApoB and Uses Thereof

Patent Number: 7,723,317

Filed: March 9, 2009

Lead Inventor: Juergen Soutschek, Alnylam Pharmaceuticals

"The invention relates to compositions and methods for modulating the expression of apolipoprotein B, and more particularly to the down-regulation of apolipoprotein B by chemically modified oligonucleotides," the patent's abstract states.


Title: iRNA Agents with Biocleavable Tethers

Patent Number: 7,723,509

Filed: Nov. 9, 2004

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

"The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group," the patent's abstract states. "The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent, coupled by way of the cleavable linking group to a targeting agent, to target cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent."


Title: microRNA Molecules

Patent Number: 7,723,510

Filed: May 11, 2007

Lead Inventor: Thomas Tuschl, Max Planck Institute

"In Caenorhabditis elegans, lin-4 and let-7 encode 22- and 21-nucleotide RNAs, respectively, that function as key regulators of developmental timing," the patent's abstract states. "Because the appearance of these short RNAs is regulated during development, they are also referred to as small temporal RNAs. We show that many more 21- and 22-nt expressed RNAs, termed microRNAs, exist in invertebrates and vertebrates, and that some of these novel RNAs, similar to let-7 stRNA, are also highly conserved. This suggests that sequence-specific post-transcriptional regulatory mechanisms mediated by small RNAs are more general than previously appreciated."


Title: Engineered microRNA Precursors for Suppressing Target Gene Expression in Plants

Patent Number: 7,723,568

Filed: Oct. 10, 2006

Lead Inventor: Linda Lutfiyya, Monsanto

The invention, the patent's abstract states, "provides molecular constructs and methods for use thereof, including constructs including heterologous miRNA recognition sites, constructs for gene suppression including a gene suppression element embedded within an intron flanked on one or on both sides by non-protein-coding sequence, constructs containing engineered miRNA or miRNA precursors, and constructs for suppression of production of mature microRNA in a cell. Also provided are transgenic plant cells, plants, and seeds containing such constructs, and methods for their use. The invention further provides transgenic plant cells, plants, and seeds containing recombinant DNA for the ligand-controlled expression of a target sequence, which may be endogenous or exogenous. Also disclosed are novel miRNAs and miRNA precursors from crop plants including maize and soy."


Title: Compositions and Methods for Inhibiting Expression of Factor VII Genes

Application Number: 20100124547

Filed: Nov. 12, 2009

Lead Inventor: Birgit Bramlage, Roche

The invention, the patent application's abstract states, "relates to a double-stranded ribonucleic acid for inhibiting the expression of a Factor VII gene. The invention also relates to a pharmaceutical composition comprising the dsRNA or nucleic acid molecules or vectors encoding the same together with a pharmaceutically acceptable carrier; methods for treating diseases caused by the expression of a Factor VII gene using said pharmaceutical composition; and methods for inhibiting the expression of Factor VII in a cell."


Title: Gold Nanorod-siRNA Complexes and Methods of Using Same

Application Number: 20100125100

Filed: Sept. 8, 2009

Lead Inventor: Earl Bergey, State University of New York, Buffalo

The patent application, its abstract states, claims "methods and compositions for inhibiting expression of one or more target genes. The compositions contain RNA polynucleotides that can inhibit expression of a target gene via RNA interference [and are] electrostatically complexed with surface functionalized gold nanorods. The RNA polynucleotides are not covalently bound to the surface functionalized GNRs.

"The method involves inhibiting expression of a target gene in an individual," the abstract notes. "The method is performed by administering to the individual an effective amount of a composition containing surface functionalized GNRs electrostatically complexed with RNA polynucleotides, such as siRNA, that can inhibit expression of the target gene via RNAi. The siRNA is not covalently bound to the surface functionalized GNRs."


Title: Methods and Compositions for Modulating the miRNA Pathway

Application Number: 20100125919

Filed: Jan. 18, 2008 PCT Filed: Jan. 18, 2008

Lead Inventor: Lionel Navarro, Centre National de la Recherche Scientifique

"A specific spectrum of plant and animal miRNAs confer enhanced resistance to virulent pathogens," according to the patent application's abstract. "Plants deficient in miRNA accumulation are hyper-susceptible to non-virulent bacterial pathogens, and virulent bacteria suppress miRNA pathways by means of injected type-III secreted proteins. Methods and compositions for modulating the miRNA pathway in plants and animals are disclosed whereby adverse effects on plant and animal development are avoided or minimized."

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