Abstract: The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications. Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules capable of mediating RNA interference (RNAi) against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.
Type:
Grant
Filed:
February 16, 2007
Date of Patent:
September 30, 2014
Assignee:
Sirna Therapeutics, Inc.
Inventors:
James McSwiggen, Bharat Chowrira, Leonid Beigelman, Dennis Macejak, Shawn Zinnen, Pamela Pavco, Peter Haeberli, David Morrissey, Kathy Fosnaugh, Sharon F Jamison, Nassim Usman, James Thompson, Chandra Vargeese, Weimin Wang, Tongqian Chen, Narendra K Vaish
Abstract: The present invention provides an Influenza A H1N1 subtype-specific aptamer. The aptamer was selected in vitro using SELEX and a microfluidic chip system. The aptamer is stable, establishing sensitivity about 100 times higher than antibody and high specificity to Influenza A H1N1 subtype. Thus, the aptamer can be effective in detection of H1N1 influenza virus.
Type:
Grant
Filed:
February 20, 2014
Date of Patent:
September 16, 2014
Assignee:
National Tsing Hua University
Inventors:
Gwo-Bin Lee, Tong-Minn Liou, Chih-Hung Wang, Hsien-Chih Lai
Abstract: The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of CTNNB1 gene expression and/or activity, and/or modulate a beta-catenin gene expression pathway. Specifically, the invention relates to double-stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against CTNNB1 gene expression.
Type:
Grant
Filed:
August 6, 2013
Date of Patent:
September 16, 2014
Assignee:
Sirna Therapeutics, Inc.
Inventors:
Duncan Brown, James J. Cunningham, Marian Gindy, Victoria Pickering, Matthew G. Stanton, Steven M. Stirdivant, Walter R. Strapps
Abstract: The invention provides methods of treating certain blood related disorders, in particular, thrombocytopenia and anemia comprising increasing miR-150 expression or inhibiting miR-150 in progenitor cells respectively.
Type:
Grant
Filed:
August 21, 2013
Date of Patent:
September 9, 2014
Assignees:
The General Hospital Corporation, Dana-Farber Cancer Institute, Massachusetts Institute of Technology
Inventors:
Jun Lu, Shangqin Guo, Benjamin Ebert, David Scadden, Todd Golub
Abstract: A miRNA expression vector including SEQ ID NO. 11. The vector is capable of improving the fertility of animals by inhibiting the expression of inhibin.
Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a Hepatitis B Virus 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 Hepatitis B Virus infection using said pharmaceutical composition; and methods for inhibiting the expression of a Hepatitis B Virus gene in a cell.
Type:
Grant
Filed:
June 28, 2012
Date of Patent:
August 19, 2014
Assignee:
Arrowhead Madison Inc.
Inventors:
Daniel Chin, Jochen Deckert, Markus Hossbach, Matthias John
Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the PCSK9 gene (PCSK9 gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the PCSK9 gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier and method for treating diseases caused by PCSK9 gene expression.
Type:
Grant
Filed:
May 15, 2012
Date of Patent:
August 19, 2014
Assignee:
Alnylam Pharmaceuticals, Inc
Inventors:
Pamela Tan, Birgit Bramlage, Maria Frank-Kamenetsky, Kevin Fitzgerald, Akin Akinc, Victor E. Kotelianski
Abstract: A method of decreasing the expression of LIM kinase 1 in a cancer cell comprising; providing an oligonucleotide consisting of the sequence of SEQ ID NO: 1; providing a cancer cell comprising an mRNA encoding LIM kinase 1; and introducing the oligonucleotide into the cancer cell, wherein the oligonucleotide decreases the expression of LIM kinase 1 in the cancer cell. The method also provides compositions of an antisense RNA LIM kinase 1 that can be administered to an individual for the purpose of inhibiting a protein kinase pathway and which further comprises methods for treating and monitoring the proliferation and metastasis of cancer cells. A kit may be used in the detection and treatment of cancer.
Type:
Grant
Filed:
April 4, 2013
Date of Patent:
August 12, 2014
Assignee:
University of Central Florida Research Foundation, Inc.
Abstract: The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of the HAMP gene (HAMP gene), comprising an antisense strand having a nucleotide sequence which is less that 30 nucleotides in length, generally 19-25 nucleotides in length, and which is substantially complementary to at least a part of the HAMP gene. The invention also relates to a pharmaceutical composition comprising the dsRNA together with a pharmaceutically acceptable carrier; methods for treating diseases caused by HAMP gene expression and the expression of the HAMP gene using the pharmaceutical composition.
Type:
Grant
Filed:
May 23, 2013
Date of Patent:
July 29, 2014
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Tomoko Nakayama, Anke Geick, Pamela Tan, Herbert Y. Lin
Abstract: The present invention relates to USP47 (ubiquitin specific protease 47) inhibitors and methods for inducing apoptosis or cell death in a target cell. In certain embodiments, the invention relates to methods and kits to screen for related agents that induce apoptosis. Additionally, the invention relates to assays for screening compounds capable of acting as USP47 inhibitors.
Type:
Grant
Filed:
November 5, 2012
Date of Patent:
July 15, 2014
Assignee:
New York University
Inventors:
Michele Pagano, Jeffrey R. Skaar, Angelo Peschiaroli, N. Valerio Dorrello
Abstract: Provided herein are isolated genomic polynucleotide fragments from the from the p15 region of chromosome 11 encoding human and tumor suppressing subtransferable candidate 4 (TSSC4) and methods of use.
Abstract: Described herein are compositions and methods for modulation of p53-dependent cell death and cell proliferation. The compositions are microRNAs and associated nucleic acids.
Type:
Grant
Filed:
February 26, 2008
Date of Patent:
July 1, 2014
Assignees:
Rosetta Genomics Ltd., Yeda Research and Development Company Ltd.
Abstract: A method for treating a patient suffering from multiple sclerosis, particularly a relapsing form of multiple sclerosis, comprising periodically administering a pharmaceutical composition comprising a therapeutically effective amount of OLIGONUCLEOTIDE 1 to the patient, thereby treating the patient.
Abstract: Cdc25A is herein identified as a substrate for ?-TrCP1- or ?-TrCP2-mediated ubiquitination and subsequent degradation via the ubiquitin-proteasome pathway. In particular, it has been found that interfering with ?-TrCP expression or function, or increasing ?-TrCP degradation, leads to accumulation of Cdc25A in a cell. Since degradation of Cdc25A is a key feature of the response to DNA damage, leading to a stall in the cell cycle during which the cell can repair the damage, Cdc25A accumulation can abolish this response, thereby sensitizing the cell to DNA damage. Described herein are assays for identifying ?-TrCP inhibitors, and method of using such inhibitors for modulating Cdc25A degradation, sensitization of tumor cells, and as adjuvants in cancer therapy based on DNA damaging agents.
Abstract: Toll-like receptor 2 (TLR2) has been found to mediate certain effects of HSV infection, particularly in neonates. Compounds that decrease TLR2 expression or activity are useful for ameliorating such deleterious effects.
Type:
Grant
Filed:
January 21, 2005
Date of Patent:
June 10, 2014
Assignee:
University of Massachusetts
Inventors:
Evelyn A. Kurt-Jones, Robert W. Finberg
Abstract: The present invention relates to methods and compositions for the treatment of diseases, including cancer, infectious diseases and autoimmune diseases. The present invention also relates to methods and compositions for improving immune function. More particularly, the present invention relates to multifunctional molecules that are capable of being delivered to cells of interest for the treatment of diseases and for the improvement in immune function.
Type:
Grant
Filed:
September 9, 2011
Date of Patent:
June 10, 2014
Assignee:
City of Hope
Inventors:
Hua Yu, Marcin Kortylewski, Richard Jove, Piotr Marek Swiderski, John J. Rossi
Abstract: MicroRNAs (miRNAs) that sensitize cancer cells to Bcl-2 family protein inhibitors are identified and described. Oligonucleotide panels, arrays and methods using the sensitizing miRNAs are also disclosed.
Type:
Grant
Filed:
December 23, 2010
Date of Patent:
June 3, 2014
Assignee:
AbbVie Inc.
Inventors:
Dimitri Semizarov, Xin Lu, Lloyd T. Lam
Abstract: The present invention relates to a pharmaceutical composition for preventing or treating arthritis, including, as an active ingredient, a material which inhibits the expression of the hypoxia-inducible factor-2? (HIF-2?) gene or the activity of the HIF-2? protein. According to the present invention, the HIF-2? of the present invention increases the expression thereof in chondrocytes or tissue in which osteoarthritis is induced, and triggers the expression of various cartilage degeneration factors and the activation of mitogen-activated protein (MAP) kinase. In addition, when HIF-2? is inhibited, the expression level of cartilage degeneration factors and the phosphorylation of MAP kinase are significantly reduced by the inhibited degree of HIF-2?. Thus, the composition of the present invention may be applied to the prevention or treatment of arthritis, and may be used for the development of therapeutics for arthritis.
Type:
Grant
Filed:
August 31, 2010
Date of Patent:
June 3, 2014
Assignee:
Gwangju Institute of Science and Technology
Abstract: Disclosed herein are antisense compounds and methods for decreasing Factor 11 and treating or preventing thromboembolic complications in an individual in need thereof. Examples of disease conditions that can be ameliorated with the administration of antisense compounds targeted to Factor 11 include thrombosis, embolism, and thromboembolism, such as, deep vein thrombosis, pulmonary embolism, myocardial infarction, and stroke. Antisense compounds targeting Factor 11 can also be used as a prophylactic treatment to prevent individuals at risk for thrombosis and embolism.
Type:
Grant
Filed:
October 8, 2012
Date of Patent:
May 27, 2014
Assignee:
Isis Pharmaceuticals, Inc.
Inventors:
Susan M. Freier, Brett P. Monia, Hong Zhang, Chenguang Zhao, Jeffrey R. Crosby, Andrew M. Siwkowski