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IP Update: Jun 10, 2010

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Title: Method for Expression of Small RNA Molecules Within a Cell

Patent Number: 7,732,193

Filed: Sept. 13, 2002

Lead Inventor: Carlos Lois-Caballe, California Institute of Technology

"The invention provides methods and compositions for the expression of small RNA molecules within a cell using a lentiviral vector," the patent's abstract states. "The methods can be used to express double-stranded RNA complexes … [as well as] small interfering RNA … which are capable of down regulating the expression of a target gene through RNA interference. A variety of cells can be treated according to the methods of the invention including embryos [and] embryogenic stem cells, allowing for the generation of transgenic animals or animals constituted partly by the transduced cells that have a specific gene or a group of genes down-regulated."


Title: Method for Expression of Small Antiviral RNA Molecules Within a Cell

Patent Number: 7,732,207

Filed: March 21, 2007

Lead Inventor: Xiao-Feng Qin, California Institute of Technology

"In one aspect, the invention provides methods and compositions for the expression of small RNA molecules within a cell using a retroviral vector," the patent's abstract states. "The methods can be used to express double-stranded RNA complexes … [as well as] small interfering RNA … that interfere with a viral life cycle by down regulating either the viral genome, a viral genome transcript, or a host cell. In another aspect, the invention provides methods for treating patients having suffering from infection, particularly infection with HIV."

Additionally, "the invention provides methods for producing siRNA encoding lentivirus where the siRNA activity may interfere with the lentiviral life cycle," the abstract adds.


Title: Methods and Compositions for RNA Interference Using Recombinant Dicer and Argonaute

Patent Number: 7,732,417

Filed: May 16, 2001

Lead Inventor: David Beach, Cold Spring Harbor Laboratory

The invention, the patent's abstract states, "provides methods for attenuating gene expression in a cell using gene-targeted double-stranded RNA. The dsRNA contains a nucleotide sequence that hybridizes under physiologic conditions of the cell to the nucleotide sequence of at least a portion of the gene to be inhibited."


Title: RNAi-Mediated Inhibition of Tumor Necrosis Factor-Alpha-Related Conditions

Patent Number: 7,732,421

Filed: May 17, 2007

Lead Inventor: John Yanni, Alcon

"RNA interference is provided for inhibition of tumor necrosis factor-alpha by silencing TNF-alpha cell-surface receptor TNF receptor-1 mRNA expression, or by silencing TNF-alpha-converting enzyme mRNA expression," the patent's abstract states. "Silencing such TNF-alpha. targets, in particular, is useful for treating patients having a TNF-alpha-related condition or at risk of developing a TNF-alpha-related condition, such as the ocular conditions dry eye, allergic conjunctivitis, or ocular inflammation, or such as dermatitis, rhinitis, or asthma."


Title: Compositions, Devices, and Methods for Treatment of Huntington's Disease Through Intracranial Delivery of siRNA

Patent Number: 7,732,591

Filed: Aug. 8, 2006

Lead Inventor: William Kaemmerer, Medtronic

The invention provides "devices, small interfering RNAs, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein," according to the patent's abstract. The invention also provides "valuable small interfering RNA vectors, systems, and methods for treating Huntington's disease in vivo without impairment of cell endoplasmic reticulum, spontaneous motor activity, or locomotor activity of a patient."


Title: Methods and Compositions for Controlling Efficacy of RNA Silencing

Patent Number: 7,732,593

Filed: June 13, 2008

Lead Inventor: Phillip Zamore, University of Massachusetts Medical School

"Based at least in part on an understanding of the mechanisms by which small RNAs mediate RNA silencing in plants, rules have been established for determining, for example, the degree of complementarity required between an RNAi-mediating agent and its target, i.e., whether mismatches are tolerated, the number of mismatches tolerated, [and] the effect of the position of the mismatches," the patent's abstract states. "Such rules are useful, in particular, in the design of improved RNAi-mediating agents which allow for more exact control of the efficacy of RNA silencing."


Title: Compositions and Methods for siRNA Inhibition of HIF-1 Alpha

Application Number: 20100136101

Filed: Dec. 3, 2009

Lead Inventor: Samuel Reich, University of Pennsylvania (Opko Health)

The invention, the patent application's abstract states, relates to the use of "small interfering RNAs [that] target HIF-1 alpha mRNA [to] inhibit expression of the HIF-1 alpha gene. 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, particularly in diseases such as diabetic retinopathy, age-related macular degeneration, and many types of cancer."


Title: Polyvalent RNA-Nanoparticle Compositions

Application Number: 20100136682

Filed: Nov. 24, 2009

Lead Inventor: Chad Mirkin, Northwestern University

The invention, the patent application's abstract states, "concerns nanoparticles functionalized with duplex RNA for a variety of uses, including but not limited to gene regulation. More specifically, the disclosure provides a new strategy for conjugating RNA to a nanoparticle to achieve increased stability and activity."


Title: Double-Stranded Oligonucleotides

Application Number: 20100136695

Filed: Nov. 3, 2008

Inventor: Tod Woolf, Invitrogen (Life Technologies)

The patent application, its abstract states, claims "antisense sequences, including duplex RNAi compositions, [that] possess improved properties over those taught in the prior art. … The invention provides optimized antisense oligomer compositions and method for making and using the both in in vitro systems and therapeutically. The invention also provides methods of making and using the improved antisense oligomer compositions."


Titles: RNA Interference-Mediated Inhibition of Cyclic Nucleotide Type 4 Phosphodiesterase Gene Expression Using Short Interfering Nucleic Acid

Application Numbers: 20100137405, 20100137406

Filed: May 2, 2008 PCT Filed: May 2, 2008

Lead Inventor: Vasant Jadhav, Merck

The patent applications, their abstracts state, relate to "compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of cyclic nucleotide type 4 phosphodiesterase gene expression and/or activity, including PDE4B1, PDE4B2, and PDE4B3 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 cyclic nucleotide type 4 phosphodiesterase gene expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases, and conditions, including but not limited to IL-6, IL-I, IL-8, IL-15, TNF-alpha, and matrix metalloproteinases such as MMP-1, MMP-2, MMP-3, MMP-9, and MMP-12," the abstracts add.

"Specifically, the [inventions relate] to double-stranded nucleic acid molecules including small nucleic acid molecules … capable of mediating RNA interference against cyclic nucleotide type 4 phosphodiesterase gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such small nucleic acid molecules," they state. The invention also relates to "small nucleic acid molecules … that can inhibit the function of RISC to modulate PDE4B gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs. … Such small nucleic acid molecules are useful, for example, in providing compositions to prevent, inhibit, or reduce inflammatory, respiratory, and autoimmune diseases, traits, and conditions, and/or other disease states associated with PDE4B gene expression or activity in a subject or organism."


Title: Single-Chain Circular RNA and Method of Producing the Same

Application Number: 20100137407

Filed: May 9, 2008 PCT Filed: May 9, 2008

Lead Inventor: Hiroshi Abe, Otsuka Pharmaceutical

The invention, the patent application's abstract states, relates to "a single-chain circular RNA having a sustained or slow-releasing RNA interference effect … [with the molecule comprising] a sense strand sequence [and] an antisense strand sequence complementary to the sense strand sequence, [as well as] identical or different two loop sequences between the sense strand and the antisense strand [that connects] both strands, wherein the sense strand and the antisense strand are paired to form a stem."


Title: Oncogenic ALL-1 Fusion Proteins for Targeting Drosha-Mediated microRNA Processing

Application Number: 20100137410

Filed: June 13, 2008 PCT Filed: June 13, 2008

Inventor: Carlo Croce, Ohio State University

The patent application, its abstract states, claims "compositions and methods for reducing the proliferation of ALL cancer cells through targeted interactions with ALL1 fusion proteins."


Title: Ras-Mediated Epigenetic Silencing Effectors and Uses Thereof

Application Number: 20100137411

Filed: Aug. 5, 2008 PCT Filed: Aug. 5, 2008

Lead Inventor: Michael Green, University of Massachusetts

"The invention relates to methods for inhibiting gene silencing, methods for inhibiting cell proliferation, methods for inhibiting Ras-mediated tumor growth, methods for screening for regulators of FAS expression, and methods for identifying inhibitors of Ras-mediated tumor growth," the patent application's abstract states.


Title: microRNAome

Application Number: 20100137413

Filed: Feb. 16, 2007 PCT Filed: Feb. 16, 2007

Lead Inventor: Jordan Cummins, Johns Hopkins University

According to the patent application's abstract, "microRNAs are a class of small non-coding RNAs that have important regulatory roles in multicellular organisms. The public miRNA database contains 321 human miRNA sequences, 234 of which have been experimentally verified. To explore the possibility that additional miRNAs are present in the human genome, we have developed an experimental approach called miRNA serial analysis of gene expression and used it to perform the largest experimental analysis of human miRNAs to date.

"Sequence analysis of 273,966 small RNA tags from human colorectal cells allowed us to identify 200 known mature miRNAs, 133 novel miRNA candidates, and 112 previously uncharacterized miRNA* forms," it adds. "To aid in the evaluation of candidate miRNAs, we disrupted the Dicer locus in three human colorectal cancer cell lines and examined known and novel miRNAs in these cells. The miRNAs are useful to diagnose and treat cancers."

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