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IP Update: Feb 3, 2011

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Title: Interfering RNA Delivery System and Uses Thereof

Patent Number: 7,879,813

Filed: Dec. 18, 2008

Inventor: Jon Chatterton, Alcon

"The invention provides a delivery system comprising a cell penetrating peptide, a polyarginine peptide, and an interfering RNA molecule," the patent's abstract states. "The system can be used for delivering interfering RNA molecules into a cell in vivo or in vitro. Therapeutic uses for the delivery system are also provided."


Title: RNA Interference-Mediating Small RNA Molecules

Application Number: 20110020234

Filed: Sept. 10, 2010

Lead Inventor: Thomas Tuschl, Max Planck Institute

"Double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference," according to the patent application's abstract. "Using a Drosophila in vitro system, we demonstrate that 19-23 [nucleotide long] short RNA fragments are the sequence-specific mediators of RNAi. The short interfering RNAs are generated by an RNase III-like processing reaction from long dsRNA. Chemically synthesized siRNA duplexes with overhanging 3' ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA.

"Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced [small interfering ribonucleoprotein particle] complex," it adds.


Title: Compositions and Methods for Inhibiting Expression of Glucocorticoid Receptor Genes

Application Number: 20110020300

Filed: May 14, 2010

Lead Inventor: Jacques Bailly, Genentech

"This invention relates to a double-stranded ribonucleic acid for inhibiting the expression of a GCR gene," the patent application's abstract states. "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 GCR gene using said pharmaceutical composition; and methods for inhibiting the expression of GCR in a cell."


Title: Compositions for Delivery of Cargo Such as Drugs, Proteins, and/or Genetic Materials

Application Number: 20110020433

Filed: Feb. 9, 2009

Inventor: Casey Cunningham, Baylor University Medical Center

The invention comprises "compositions and methods for delivery of cargo molecules to a patient or subject in need thereof [and which include] a proteoliposome carrier vehicle that incorporates an RLIP76 protein and contains the cargo molecule," the patent application's abstract states. "The vehicle effectively delivers the cargo molecule systemically throughout the tissues of the body including into the central nervous system."


Title: Precursor miRNA Loop-Modulated Target Regulation

Application Number: 20110020816

Filed: May 28, 2010

Lead Inventor: Chang-Zheng Chen, Stanford University

"By modifying nucleotides from either or both the stem and loop of precursor-miRNA, greater specificity is achieved as to the mRNAs targeted," the patent application's abstract states. "The improved efficiency in target regulation can be either mediated by direct base-pairings between targets and precursor miRNAs or indirectly by energy constraints on the availability of such base-pairings. It is found that pri- and pre-miRNA are active in the absence of functional mature miRNA, so pri- or pre-miRNA or truncated portion thereof, but not as mature miRNA, can be used as RNAi agents by themselves.

"Furthermore, besides the seed sequence of the stem of the pre-miRNA and 3'-extensions thereof to provide greater complementarity to the target mRNA, nucleotides in the loop can affect the activity and specificity of the precursor-miRNA and the processing and binding to target mRNA," the abstract adds. "By using mutated sequences in the natural pri- or pre-miRNA or modified mimetics, one can screen for target mRNA, target a unique mRNA or a group of mRNAs for regulation, and modulate cell properties with greater specificity and investigate cellular activity as to phenotype and response to external stimuli in the presence or absence of at least partial target protein expression."


Title: siRNA Targeting Amyloid Beta Precursor Protein

Application Number: 20110021382

Filed: Sept. 20, 2010

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

"Efficient sequence-specific gene silencing is possible through the use of siRNA technology," the patent application'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 APP."


Title: Novel Nucleic Acid

Application Number: 20110021600

Filed: Sept. 4, 2007

Lead Inventor: Yoji Yamada, Kyowa Hakko Kirin

The invention, the patent application's abstract states, comprises a "novel nucleic acid, a vector that expresses the nucleic acid, a screening method for a substance that controls the nucleic acid, a method of detecting the expression and a mutation of the nucleic acid, and a diagnostic method or therapeutic method for a disease caused by an abnormality of mesenchymal stem cells, mast cells or the like, or a disease caused by a cell proliferation abnormality, such as cancer."


Title: Composition Containing microRNA-21 Inhibitor for Enhancing Radiation Sensitivity

Application Number: 20110021601

Filed: July 30, 2008

Lead Inventor: Jong Bae Park, National Cancer Center (Korea)

The invention, the patent application's abstract states, comprises a "radiation sensitivity-enhancing composition in which a microRNA-21 inhibitor acts as an active ingredient. The microRNA-21 inhibitor is an antisense nucleic acid molecule binding complementarily to microRNA-21. The composition can be administered to a patient in conjunction with irradiation. The inhibitor can act as a radiosensitizer, enhancing the therapeutic effect of such irradiation on cancer high in microRNA-21 expression level, particularly, glioma."


Title: Methods and Compositions for the Specific Inhibition of KRAS by Asymmetric Double-Stranded RNA

Application Number: 20110021604

Filed: April 5, 2010

Inventor: Bob Brown, Dicerna Pharmaceuticals

"This invention relates to compounds, compositions, and methods useful for reducing KRAS target RNA and protein levels via use of Dicer-substrate siRNA agents possessing asymmetric end structures."


Title: Means and Methods for the Specific Inhibition of Genes in Cells and Tissue of the CNS and/or Eye

Application Number: 20110021605

Filed: July 1, 2010

Lead Inventor: Ralf Wilhelm Schulte

The invention, the patent application's abstract states, comprises a "method for the specific modulation of the expression of target genes in cells and/or tissues of the CNS and/or eye, wherein a composition comprising one or more doubled stranded oligoribonucleotides is introduced into the cell, tissue, or organism outside the blood-brain or blood-retina barriers.

"Furthermore, a method for the identification and validation of the function of a gene is provided, wherein the method provides a test cell, test tissue, or test organism, which allow information to be gained on the function of the target gene. In addition, compositions and kits are described useful for those methods."


Title: RNAi Modulation of RSV, PIV, and Other Respiratory Viruses and Uses Thereof

Application Number: 20110021606

Filed: July 23, 2010

Inventor: Sailen Barik, Alnylam Pharmaceuticals

The invention, the patent application's abstract states, is "based on the in vivo demonstration that RSV and PIV can be inhibited through intranasal administration of RNAi agents, as well as by parenteral administration of such agents. Further, it is shown that effective viral reduction can be achieved with more than one virus being treated concurrently. Based on these findings, the … invention provides general and specific compositions and methods that are useful in reducing RSV or PIV mRNA levels, RSV or PIV protein levels, and viral titers in a subject."


Title: Methods and Compositions Relating to Carcinoma Stem Cells

Application Number: 20110021607

Filed: Jan. 30, 2009

Lead Inventor: Michael Clarke, Stanford University

The invention, the patent application's abstract states, relates to "microRNA markers of breast cancer stem cells.

"The markers are polynucleotides that are differentially expressed in BCSC as compared to normal counterpart cells," it adds. "Uses of the markers include use as targets for therapeutic intervention, as targets for drug development, and for diagnostic or prognostic methods relating to breast cancer and BCSC cell populations. BCSCs have the phenotype of having lower expression of certain miRNAs compared to normal breast epithelial cells, or to cancer cells that are not cancer stem cells."


Title: Methods for Nucleic Acid Transfer into Cells

Application Number: 20110021608

Filed: Oct. 12, 2010

Lead Inventor: David Dean, Northwestern University

The invention, the patent application's abstract states, comprises "methods for increasing the transfer of nucleic acids into cells. In particular, the … invention provides for the use of inhibitors of HDAC6, a cytoplasmic histone deacetylase present in mammalian cells by, for example, small molecules or siRNA treatment, in increasing gene transfer and/or expression in cells in vitro and in vivo for research and gene therapy applications."


Title: microRNA Signatures Associated with Cytogenetics and Prognosis in Acute Myeloid Leukemia and Uses Thereof

Application Number: 20110021609

Filed: Feb. 27, 2009

Lead Inventor: Carlo Croce, Ohio State University

The invention comprises "methods and compositions utilizing an miRNA signature for the diagnosis, prognosis, and/or treatment of leukemia associated diseases, particularly acute myeloid leukemia," the patent application's abstract states.

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