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IP Update: Sep 3, 2009

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Title: Vectors and Methods for High-Throughput Co-Expression

Number: 7,582,475

Filed: Jan. 6, 2006

Lead Inventor: Peter Horanyi, University of Georgia

The invention, the patent’s abstract states, comprises RNAi “vectors and methods for high-throughput co-expression.”


Title: Conditional Disruption of Dicer1 in Cell Lines and Non-Human Mammals

Number: 7,582,741

Filed: July 26, 2005

Lead Inventor: Stephen Jones, University of Massachusetts

The invention “is based, at least in part, on the generation of cells, cell lines, and non-human mammals that contain a synthetic conditional allele of the Dicer1 gene,” the patent’s abstract states. “Many of the constructs, methods, animals, and cells of the invention feature recombinase recognition sites positioned on either flank of a sequence that comprises exons 14, 15, and 16 of the Dicer1 gene. Through readily manipulable introduction, administration, or expression of a recombinase, exons 14 through 16 of Dicer1 may be [directly] excised, producing a functional knockout of Dicer1 in cells containing these recombinase-induced alleles of the invention.”


Title: Chemically Modified Oligonucleotides

Number: 7,582,744

Filed: Aug. 10, 2005

Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals

The invention relates to “composition and methods for making and using chemically modified oligonucleotides agents for inhibiting gene expression,” according to the patent’s abstract.

The patent adds that the invention particularly relates to making and using single-stranded, chemically modified oligos capable of inhibiting gene expression.


Title: Compositions and Methods for Inhibiting Expression of Nav1.8 Gene

Number: 7,582,745

Filed: Nov. 3, 2006

Lead Inventor: Dinah Sah, Alnylam Pharmaceuticals

“The invention relates to a double-stranded ribonucleic acid for inhibiting the expression of the Nav1.8 gene, comprising an antisense strand having a nucleotide sequence which is less that 25 nucleotides in length and which is substantially complementary to at least a part of the Nav1.8 gene,” the patent’s abstract states. “The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier, methods for treating diseases caused by the expression of the Nav1.8 gene using the pharmaceutical composition, and methods for inhibiting the expression of the Nav1.8 gene in a cell.”


Title: siRNA Targeting Complement Component 3

Number: 7,582,746

Filed: Oct. 26, 2007

Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)

“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent’s abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed, including those directed to nucleotide sequences for C3.”


Title: siRNA Targeting Inner Centromere Protein Antigens

Number: 7,582,747

Filed: June 6, 2008

Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)

“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” according to the patent’s abstract. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed, including those directed to nucleotide sequences for INCENP.”


Title: Recombinant Vector Expressing MDR1 shRNA and Thymidine Kinase and Use Thereof

Number: 20090214435

Filed: March 18, 2008

Lead Inventor: In San Kim, Kyungpook National University

The invention, the patent application’s abstract states, comprises “a recombinant vector capable of expressing MDR1 shRNA and thymidine kinase, and a use thereof.

“More specifically, [the invention involves] a recombinant vector capable of efficiently expressing MDR1 shRNA and thymidine kinase in a host cell; a transfectant cell comprising the same recombinant vector; a composition for treating neoplastic diseases, comprising the same recombinant vector; and a method for imaging of neoplastic lesions using the same recombinant vector,” the abstract adds. “The recombinant vector … is capable of achieving efficient intracellular expression of MDR1 shRNA and a thymidine kinase-GFP fusion protein within the host cell and is therefore highly effective for combined therapy of anticancer drugs. Further, the recombinant vector … enables imaging of neoplastic lesions … [and] can be used in combination with other anticancer drugs for treatment of neoplastic diseases.”


Title: Inhibitors of RegIII Proteins as Asthma Therapeutics

Number: 20090214516

Filed: June 3, 2005 PCT Filed: June 3, 2005

Lead Inventor: Maximillian Follettie, Wyeth

The patent application, its abstract states, claims “methods of screening for agents for treating asthma. … The methods involve screening for agents that decrease the production or activity of a RegIII protein that has been discovered herein to play a role in producing the symptoms and pathological complications involved in asthma. Methods of treating asthma, as well as screening for and treating with inhibitors of a RegIII protein are also provided.”

The application specifically claims the use of an RNAi agent to decrease the production of RegIII protein.


Title: Novel Gene Therapy Approach for Treating the Metabolic Disorder Obesity

Number: 20090214637

Filed: April 30, 2009

Inventor: Sergey Musatov, Neurologix

The invention relates to “novel methods and compositions for treating metabolic disorders, the patent application’s abstract states. “Some aspects pertain to the use of gene therapy to treat diseases related to metabolic disorders, such as diabetes, obesity, high blood pressure, wasting syndrome, cachexia, and atherogenic dyslipidemia.”

The invention also relates to “the use of vectors such as a recombinant adeno-associated virus to deliver at least a portion of a gene that can increase or decrease expression of a therapeutic protein of interest, e.g., in cells in a specific region of the brain associated with metabolic disorder,” the abstract notes. The application also “discloses the use of vectors such as a recombinant adeno-associated virus for the delivery of small [interfering] RNAs capable of decreasing expression of a deleterious protein involved in the disorder.”


Title: Novel Lipids for Transfection of Nucleic Acids

Number: 20090215067

Filed: April 27, 2009

Lead Inventor: Gulilat Gebeyehu, Molecular Transfer

The invention, the patent application’s abstract states, comprises “cationic lipid compositions … that are useful for efficient delivery of macromolecules, such as nucleic acids, into a wide variety of eukaryotic cell types. Methods for using the compositions also are provided.”


Title: RNA Interference-Induction Element and Use Thereof

Number: 20090215644

Filed: May 15, 2006 PCT Filed: May 15, 2006

Lead Inventor: Kojiro Ishii, Kurume University

The invention comprises “an RNA interference induction element containing a nucleotide sequence selected from among [specific] nucleotide sequences,” which can be used to “knock down a desired target gene and to produce a siRNA for a desired target gene.”


Title: Screening and Therapeutic Methods for NSCLC-Targeting CDCA1-KNTC2 Complex

Number: 20090215683

Filed: July 19, 2006 PCT Filed: July 19, 2006

Lead Inventor: Yusuke Nakamura, Oncotheraphy Science

The invention is “based on the observation that the co-activation of CDCA1 and KNTC2, and their cognate interactions, play significant roles in lung-cancer progression and that methods of inhibiting the complex can be used to treat non-small-cell lung cancer,” the patent application’s abstract states.

The application specifically claims the use of a dsRNA, including an siRNA, capable of inhibiting KNTC2.


Title: Compositions and Methods for Regulating Gene Transcription

Number: 20090215860

Filed: July 17, 2005 PCT Filed: July 17, 2005

Lead Inventor: Kevin Morris, Scripps Research Institute

“The invention is directed to compositions and methods for RNA-mediated gene regulation, [such as] transcription regulation by transcriptional silencing of genes,” the patent application’s abstract states. “In one aspect, the invention provides methods using siRNAs directed at a transcription regulator, [for instance] a promoter or enhance sequence, of a gene target molecule. In one aspect, this results in in vivo DNA methylation and/or modification of associated chromatin accompanied by and/or partial or complete transcription gene silencing in a cell, such as a mammalian cell.”


Title: microRNA

Number: 20090215862

Filed: April 12, 2006 PCT Filed: April 12, 2006

Lead Inventor: Cesare Peschle, Istituto Superiore di Sanita

According to the patent application’s abstract, a “microRNA capable of interacting with the 3' untranslated region of kit protein mRNA is useful in treating kit-dependent tumors, and inhibitors therefore are useful in treating suppressed hematopoiesis in cancer patients or abnormal erythropoiesis in beta thalassemia, for example.”


Title: Gene Silencing of Protease Activated Receptor 1

Number: 20090215863

Filed: Aug. 17, 2006 PCT Filed: Aug. 17, 2006

Inventor: Rachel Bar-Shavit, Hadassah University (InCure)

The invention, the patent application’s abstract states, “relates to nucleic acid molecules, vectors, compositions, and methods useful for modulating protease-activated receptor 1 gene expression via RNA interference. In particular, the … invention features small interfering RNA and short hairpin RNA molecules and methods for modulating the expression of protease-activated receptor 1 gene.”


Title: Oligoribonucleotide Inhibitors of Nrf2 and Methods of Use Thereof for Treatment of Cancer

Number: 20090215864

Filed: March 19, 2008

Inventor: Elena Feinstein, Quark Pharmaceuticals

“The invention provides novel double-stranded oligoribonucleotides that inhibit the Nrf2 gene,” the patent application’s abstract states. “The invention also provides a pharmaceutical composition comprising one or more such oligoribonucleotides, and a vector capable of expressing the oligoribonucleotide. [It] also relates to methods and compositions for treating or preventing the incidence or severity of a cancerous disease, particularly various lung cancers.”


Title: Nucleic Acid Molecules and Collections Thereof, Their Application, and Identification

Number: 20090215865

Filed: Jan. 10, 2006 PCT Filed: Jan. 10, 2006

Lead Inventor: Ronald Plasterk, Hubrecht Laboratory

“The invention provides a method for characterizing a sample comprising nucleic acid derived from a cell,” the patent application’s abstract states. “The method comprises determining whether a sample comprises at least a minimal sequence of at least one new microRNA, according to the invention or a mammalian ortholog thereof, and characterizing the sample on the basis of the presence or absence of the miRNA. The invention further provides new nucleic acid molecules and collections thereof and their use in therapeutic and diagnostic applications, [as well as] … a method for identifying a miRNA molecule or a precursor molecule thereof.”


Title: Cancer Cell-Specific Cell Proliferation Inhibitors

Number: 20090215867

Filed: Nov. 17, 2005 PCT Filed: Nov. 17, 2005

Lead Inventor: Motoki Takagi, GeneCare Research Institute

“Although suppressing expression of the RecQ1 gene, a RecQ helicase family gene, shows suppressive effects on cell proliferation in cancer cells, such effects are not observed in human TIG3 cells, which are normal cells,” the patent application’s abstract states. “Hence, the … inventors discovered that siRNAs against RecQ1 gene have cancer cell-specific cell proliferation-suppressing effects that are mediated by suppression of the expression of said gene.”


Title: Methods for Controlling Stem Cell Differentiation

Number: 20090215872

Filed: June 30, 2006 PCT Filed: June 30, 2006

Inventor: Jeannie Lee, Harvard Medical School

The invention, the patent application’s abstract states, comprises “methods for controlling stem cell differentiation through the introduction of transgenes having Xic, Tsix, or Xite sequences to block differentiation and the removal of the transgenes to allow differentiation. Also disclosed are small RNA molecules and methods for using the small RNA molecules to control stem cell differentiation, [as well as] … stem cells genetically modified by the introduction of Xic, Tsix, or Xite sequences.”


Title: Methods and Compositions for the Diagnosis and Treatment of Schizophrenia

Number: 20090215877

Filed: Nov. 3, 2008

Inventor: Linda Brzustowicz, Rutgers University

The invention, the patent application’s abstract states, comprises “compositions and methods relating to the diagnosis and treatment of neuropsychiatric disorders, such as schizophrenia, schizoaffective disorders, and bipolar disorders. … Also provided are methods for screening therapeutic agents having efficacy for the treatment of such disorders.”

The application specifically claims an siRNA that inhibits NOS1AP-NOS1AP binding protein-complex formation.


Title: Cre-lox-Based Gene-Knockdown Constructs and Methods of Use Thereof

Number: 20090217397

Filed: June 11, 2007

Inventor: Patrick Stern, Massachusetts Institute of Technology

The invention “relates to vectors, compositions, and methods for conditional, Cre-lox regulated, RNA interference,” the patent application’s abstract states. “The vectors allow for spatial and temporal control of miRNA expression in vivo.”


Title: Lentiviral Vectors that Provide Improved Expression and Reduced Variegation After Transgenesis

Number: 20090217399

Filed: March 16, 2007 PCT Filed: March 16, 2007

Inventor: Patrick Stern, Massachusetts Institute of Technology

The invention, the patent application’s abstract states, “provides new lentiviral vectors that include an anti-repressor element and, optionally, a scaffold-attachment region. The lentiviral vectors provide expression of a heterologous nucleic acid in at least 50 [percent] of the cells of multiple cell types when used for lentiviral transgenesis. In certain embodiments of the invention, the heterologous nucleic acid encodes an RNAi agent, such as an shRNA,” the abstract notes.

“The invention further provides transgenic, non-human animals generated using a lentiviral vector that includes an ARE and optional SAR,” it adds. “In addition, the invention provides a variety of methods for using the vectors including for achieving gene silencing in eukaryotic cells and transgenic animals, and methods of treating disease. The invention also provides animal models of human disease in which one or more genes is functionally silenced using a lentiviral vector of the invention.”


Title: Cell-Based RNA Interference and Related Methods and Compositions

Number: 20090217404

Filed: Feb. 23, 2008

Inventor: Scott Lowe, Cold Spring Harbor Laboratory

“The invention provides, among other things, methods for performing RNA interference in stem cells, such as embryonic stem cells, and methods for using such stem cells in vivo,” according to the patent application’s abstract. “The invention also provides various animal models based on conditional/inducible, reversible, tissue-specific/spacial, and/or developmental stage-specific/temporal RNAi of certain target genes, which animal model may be useful for … drug target identification and/or validation.”

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