Abstract: Disclosed herein are compounds, compositions and methods for modulating the expression of huntingtin in a cell, tissue or animal. Further provided are methods of slowing or preventing Huntington's disease progression using an antisense compound targeted to huntingtin. Additionally provided are methods of delaying or preventing the onset of Huntingtin's disease in an individual susceptible to Huntingtin's Disease. Also provided are uses of disclosed compounds and compositions in the manufacture of a medicament for treatment of diseases and disorders.
Type:
Grant
Filed:
December 7, 2012
Date of Patent:
June 16, 2015
Assignees:
Isis Pharmaceuticals, Inc., CHDI Foundation Inc.
Inventors:
Gene Hung, Janet M. Leeds, C. Frank Bennett, Susan M. Freier
Abstract: An object of the present invention is to provide a novel double-stranded RNA that has high resistance to nuclease and cellular uptake efficiency and that can produce an excellent RNA interference effect. The present invention provides a double-stranded lipid-modified RNA comprising an antisense strand having a nucleotide sequence complementary to a target sequence in a target gene, and a sense strand having a nucleotide sequence complementary to the antisense strand, the double-stranded RNA being capable of suppressing the expression of the target gene, and the sense strand having a double-stranded lipid bound directly or via a linker to at least one of the first to sixth nucleotides from the 5? end.
Type:
Grant
Filed:
March 31, 2009
Date of Patent:
May 26, 2015
Assignees:
NATIONAL INSTITUTE OF ADVANCED INDUSTRIAL SCIENCE AND TECHNOLOGY, OTSUKA PHARMACEUTICAL CO., LTD.
Abstract: The present invention relates functional ligands to target molecules, particularly to functional nucleic acids and modifications thereof, and to methods for simultaneously generating, for example, numerous different functional biomolecules, particularly to methods for generating numerous different functional nucleic acids against multiple target molecules simultaneously. The present invention further relates to functional ligands which bind with affinity to target molecules. The present invention further relates to methods for generating, for example, functional biomolecules, particularly to functional nucleic acids, that bind with functional activity to another biomolecule, such as a receptor molecule. More than one or multiple targets as used herein may generally include different types of targets, and/or may also include a multitude of a singular type of targets at different conditions, such as, for example, temperature, pH, chemical environment, and/or any other appropriate conditions.
Abstract: A method of decreasing NFATc2 activity in a lymphocyte includes administering to the lymphocyte an amount of an NFATc2 mRNA antagonist that binds to a binding site on the 3?UTR of NFATc2 mRNA effective to decrease the activity of NFATc2 mRNA in the lymphocyte.
Abstract: DNA aptamers are high affinity ligands selected by genetic enrichment techniques to bind to specific protein targets. Because these represent chemically stable and reproducible molecules, they have application as affinity reagents and/or therapeutic drugs to affect the target protein's actions. NF-kB is an important mediator of the innate immune response and mediator of tissue inflammation. Although RNA and double stranded DNA aptamers have been identified to bind to the NF-kB family of proteins, the present invention represents the first identification of single stranded DNA aptamers that recognize NFkB RelA. The aptamers disclosed herein bind to several distinct regions of RelA and may be useful to antagonize the DNA binding of RelA as an inhibitor of cellular inflammation, visualize the location or amount of RelA in tissues from pathological conditions, or to quantitatively measure the activated state of RelA by affinity binding.
Type:
Grant
Filed:
April 12, 2012
Date of Patent:
April 28, 2015
Assignee:
Board of Regents, The University of Texas System
Inventors:
Steven G. Widen, Thomas G. Wood, Allan R. Brasier, Yingxin Zhao
Abstract: The invention relates to compositions and methods for preventing and diminishing virus infection. The invention further relates to compositions and methods for diminishing inflammation associated with viral infection. The invention also relates to compositions and methods for interfering with TLR activation, and thereby diminishing inflammation associated with viral infection.
Type:
Grant
Filed:
December 17, 2009
Date of Patent:
April 14, 2015
Assignee:
Drexel University
Inventors:
Peter D. Katsikis, Alina C. Boesteanu, Sefik S. Alkan
Abstract: The present disclosure relates to regulation of functions of hematopoietic stem cells (HSCs) by delivering of miRNAs, including miR-125b, miR-126, and miR-155, to HSCs. For example, in some embodiments, blood output in a mammal can be increased by administering miR-125b, miR-126, and/or miR-155 oligonucleotides. Also disclosed are methods for promoting hematopoietic stem cell engraftment and method for treating a myeloproliferative disorder.
Abstract: The present invention provides method of optimizing the efficacy and potency of antisense drugs. In certain embodiments, the invention provides assays useful for determining favorable oligonucleotide characteristics and excipients for improved cellular uptake.
Abstract: This invention relates to modified double-stranded oligoribonucleic acid (dsRNA) having improved stability in cells and biological fluids, and methods of making and identifying dsRNA having improved stability, and of using the dsRNA to inhibit the expression or function of a target gene.
Type:
Grant
Filed:
March 27, 2012
Date of Patent:
March 31, 2015
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Hans-Peter Vornlocher, Ingo Roehl, Philipp Hadwiger, Tracy Stage Zimmermann, Muthiah Manoharan, Kallanthottathil G. Rajeev, Akin Akinc
Abstract: The invention discloses a kit which comprises a formulation containing artemisinin or the derivatives thereof, a formulation containing ribonuclease, and a specification.
Type:
Grant
Filed:
September 10, 2012
Date of Patent:
March 24, 2015
Assignees:
Shanghai Biomodel Organism Science & Technology Co., Ltd., Shanghai Research Center for Model Organisms
Inventors:
Qingcheng Wang, Ruling Shen, Jun Li, Jian Fei, Zhugang Wang
Abstract: A method for recycling metals is provided by using extracellular proteins excreted by a specific thermophilic bacteria strain, Tepidimonas fonticaldi sp. nov., in which the extracellular proteins show excellent metal-ion binding ability, being useful in recycling rare earth metal ions and precious metal ions from geothermal fluids, boiler solutions, industrial wastewater or hard water.
Type:
Grant
Filed:
December 30, 2013
Date of Patent:
March 3, 2015
Assignee:
Industrial Technology Research Institute
Abstract: System, including methods and apparatus, for performing a digital assay with multiplexed detection of two or more distinct targets in the same optical channel.
Type:
Grant
Filed:
July 12, 2012
Date of Patent:
February 10, 2015
Assignee:
Bio-Rad Laboratories, Inc.
Inventors:
Serge Saxonov, Simant Dube, John Frederick Regan
Abstract: The present invention, at least in part, relates to the discovery of efficacious delivery of an RNAi agent (in preferred aspects of the invention, an siRNA) to a transplantable tissue. Organ rejection, transplantation-mediated transmission of viral infection, and triggering of apoptosis in transplanted tissues can each be minimized by the methods and compositions of the instant invention. The RNAi agent(s) of the instant invention can be delivered as “naked” molecules, or using liposomal and other modes of delivery, to transplantable tissues. Such delivery can occur via perfusion of the RNAi agent in solution through the vasculature of a whole or partial organ; or tissues including transplantable cells and cell lines may be bathed, injected or otherwise treated with RNAi agents. Preferred transplantable tissues include, for example, pancreas, liver, kidney, heart, lung, and all cells and cell lines derived from such tissues (e.g., pancreatic islet cells that may, e.g.
Abstract: The present invention relates to a chromogenic method for simultaneously determining the activity of multiple coagulation proteases or for simultaneously determining the inhibition of multiple coagulation proteases in a single test reaction. For this purpose, use is made of two chromogenic substrates which have different absorption maxima and whose color signals can be separated spectrally.
Abstract: The present invention provides nucleosides and oligonucleotides comprising a 5? phosphate mimics of formula (IVc) or (Vc). One aspect of the present invention relates to modified nucleosides and oligonucleotides comprising such dinucleotide of formula (Ia). Another aspect of the invention relates to a method of inhibiting the expression of a gene in call, the method comprising (a) contacting an oligonucleotide of the invention with the cell; and (b) maintaining the cell from step (a) for a time sufficient to obtain degradation of the mRNA of the target gene.
Type:
Grant
Filed:
July 7, 2010
Date of Patent:
January 6, 2015
Assignee:
Alnylam Pharmaceuticals, Inc.
Inventors:
Muthiah Manoharan, Kallanthottathil G. Rajeev, Marija Prhavc, Ivan Zlatev
Abstract: This invention relates to nucleic acid aptamers that recognize and bind the complement protein C3 or its biologically active proteolytic products and methods of their use. Particularly preferred are bi-functional aptamer construct that binding specifically with C3b or iC3b, and another target protein. Use of these molecular constructs for commandeering the opsonization process is also described herein.
Type:
Grant
Filed:
May 20, 2009
Date of Patent:
January 6, 2015
Assignee:
The Research Foundation of State University of New York
Inventors:
Hua Shi, Albert J. T. Millis, Kimi Nishikawa, Prabhat Kumar Mallik
Abstract: The invention provides compositions and methods for reducing expression of a target gene in a cell, involving contacting a cell with an isolated double stranded nucleic acid (dsNA) in an amount effective to reduce expression of a target gene in a cell. The dsNAs of the invention possess a single stranded extension (in most embodiments, the single stranded extension comprises at least one modified nucleotide and/or phosphate back bone modification). Such single stranded extended Dicer-substrate siRNAs (DsiRNAs) were demonstrated to be effective RNA inhibitory agents compared to corresponding double stranded DsiRNAs.
Abstract: A method and point of use kit to determine under field conditions the presence and/or amount of a selected Pseudomonas species capable of bioremediating organic environmental pollutants, such as petroleum hydrocarbons.