Patents Issued in November 15, 2018
  • MIXING DEVICE FOR MIXING THE CONTENT OF A BIOREACTOR, COMPRISING A SYNCHRONIZATION MECHANISM
    Publication number: 20180327707
    Abstract: The present invention relates to a system comprising a mixing device for mixing the content of a bioreactor. The system comprises at least one movement device for initiating a mixing movement in the bioreactor or in a holder for receiving a bioreactor and at least one sensor that can be arranged in or on the bioreactor for receiving at least one physiological or physical measurement variable. The mixing device or the bioreactor further comprises a sensor or transmitter for generating a synchronization measurement variable.
    Type: Application
    Filed: November 16, 2016
    Publication date: November 15, 2018
    Inventors: Simon Brütsch, Jochen Scholz
  • Instrument resource scheduling
    Publication number: 20180327708
    Abstract: Aspects of the present invention relate to an automated cell culture instrument and to methods for operating such an instrument. In one aspect, the cell culture instrument includes a computing unit and a plurality of electrically controllable resources (e.g., sensors, actuators, etc.). The computing unit receives a protocol, schedules the resources so as to execute the protocol, and operates the resources as scheduled or in response to so as to provide an improved environment for cell culture growth. The computing unit may simulate protocol execution in connection with the scheduling or execution of the protocol.
    Type: Application
    Filed: November 18, 2016
    Publication date: November 15, 2018
    Inventors: Howard Cannon, Keith Moulton
  • A Method Of Producing High Amount Of Ethanol At High Temperature By Modified Yeast Strain Saccharomyces Cerevisiae
    Publication number: 20180327709
    Abstract: The present invention relates to a modified yeast strain of Saccharomyces cerevisiae having MCC accession number 0069 with osmo-tolerant, thermo-tolerant, ethanol tolerant and self-flocculation properties. Further, the present invention relates to a method for obtaining modified yeast strain. The present invention also relates to a method of production of ethanol at high temperature using said yeast strain. The ethanol produced by the method disclosed in the present invention is used as fuel.
    Type: Application
    Filed: August 4, 2016
    Publication date: November 15, 2018
    Inventor: Rajendra Surana
  • Process for improved fermentation of a microorganism
    Publication number: 20180327710
    Abstract: A method for improving biomass production and/or growth rate of a microorganism in a fermentation process is shown, and includes: (i) providing one or more microorganisms; (ii) providing a fermentation substrate suitable for fermenting the one or more microorganisms; (iii) mixing the one or more microorganisms and the fermentation substrate providing a fermentation broth; (iv) adding the fermentation broth to a fermentation tank; (v) injecting at least one gaseous substrate into the fermentation broth; (vi) running the fermentation process for a fermentation period of at least 1 hour; wherein the at least one gaseous substrate comprises one or more greenhouse gases, such as carbon dioxide (CO2).
    Type: Application
    Filed: November 8, 2016
    Publication date: November 15, 2018
    Applicant: UNIBO A/S
    Inventor: Subir Kumar NANDY
  • Fermentation Processes With Reduced Foaming
    Publication number: 20180327711
    Abstract: The present invention relates to processes of producing a fermentation product from readily fermentable sugar-material in a fermentation vat comprising a fermentation medium, comprising: feeding the readily fermentable sugar-material into the fermentation vat comprising a slurry of fermenting organism; fermenting the readily fermentable sugar material into a desired fermentation product, wherein S8A protease is added during or after feeding of the readily fermentable sugar-material into fermentation vat or during fermentation of the readily fermentable sugar-material into the desired fermentation product. The invention also related to the use of S8A protease for reducing foaming in the fermentation wells generating by the fermenting organism during fermentation of the readily fermentable sugar-material.
    Type: Application
    Filed: November 11, 2016
    Publication date: November 15, 2018
    Applicant: Novozymes A/S
    Inventors: Felipe Senne De Oliveira Lino, Camila Do Nascimento, João G. R. Destro, Kenneth Jensen
  • METHODS OF MAKING SPHEROIDS INCLUDING BIOLOGICALLY-RELEVANT MATERIALS
    Publication number: 20180327712
    Abstract: Methods of making a spheroid are provided whereby a suspension is first produced including one or more biologically-relevant materials dispersed within a biocompatible medium. A droplet of the suspension is then bioprinted into a salt solution by bringing the droplet into contact with a surface of the salt solution in a controlled manner to reproducibly yield a spheroid containing a desired size and a desired amount of biologically-relevant materials.
    Type: Application
    Filed: July 26, 2018
    Publication date: November 15, 2018
    Inventors: Stuart K. Williams, Jeremiah S. Touroo, James B. Hoying
  • TREATMENT OF RETINAL DEGENERATION USING PROGENITOR CELLS
    Publication number: 20180327713
    Abstract: Methods and compositions for treating and reducing retinal degeneration using progenitor cells and conditioned media from progenitor cells, such as postpartum-derived cells are disclosed. Trophic factors and other agents secreted by the progenitor cells that protect retinal cells and inhibit apoptosis of retinal cells such as photoreceptor cells are also disclosed.
    Type: Application
    Filed: July 19, 2018
    Publication date: November 15, 2018
    Applicant: JANSSEN BIOTECH, INC.
    Inventors: IAN R. HARRIS, JING CAO, NADINE DEJNEKA
  • NEURAL CELL EXTRACELLULAR VESSICLES
    Publication number: 20180327714
    Abstract: Disclosed herein are neural extracellular vesicles (EVs) and methods of using these EVs in the treatment of spinal cord injury, stroke, and traumatic brain injury and neurodegenerative diseases.
    Type: Application
    Filed: November 16, 2016
    Publication date: November 15, 2018
    Applicants: UNIVERSITY OF GEORGIA RESEARCH FOUNDATION, INC., ARUNA BIOMEDICAL, INC.
    Inventors: Steven L. Stice, Robin Lynn Webb, Tracy A. Stice
  • Engineered 3D-Printed Artificial Axons
    Publication number: 20180327715
    Abstract: Materials and methods for cell-mimetics having mechanical properties of biological neural axons are provided. A cell-mimetic device includes an array of fibers comprised of hexanediol diacrylate (HDDA) or an HDDA derivative, and at least one derivative of polyethylene glycol (PEG) selected from the group consisting of: PEG-acrylate, PEG-diacrylate, and any multi-arm PEG-acrylate.
    Type: Application
    Filed: May 9, 2018
    Publication date: November 15, 2018
    Inventors: Daniela Espinosa-Hoyos, Anna E. Jagielska, Huifeng Du, Nicholas X. Fang, Krystyn J. Van Vliet
  • MODIFIED CELLS FOR PRODUCTION OF BLOOD CELLS
    Publication number: 20180327716
    Abstract: The present disclosure relates to a genetically modified cell (e.g., stem cells) containing a complete or partial gene deletion of one or more genes of a blood group antigen (BGA) biosynthesis or transportation pathway. The systems and methods provided herein facilitate the generation of blood substitutes (e.g., blood cells).
    Type: Application
    Filed: October 25, 2016
    Publication date: November 15, 2018
    Inventor: Zhao Wu
  • Methods for Cardiac Differentiation of Human Induced Pluripotent Stem Cells
    Publication number: 20180327717
    Abstract: The present invention relates to monolayer cardiac differentiation techniques utilizing defined conditions providing feeder-free monolayer culture systems, serum-based or serum free, and applicable to both healthy control and patient derived stem cells.
    Type: Application
    Filed: August 31, 2016
    Publication date: November 15, 2018
    Applicant: The Trustees of Columbia University in the City of New York
    Inventors: Masayuki Yazawa, LouJin Song
  • ENHANCED DIFFERENTIATION OF MESENCHYMAL STEM CELLS
    Publication number: 20180327718
    Abstract: Isolated populations of mesenchymal stem cells (MSCs) are provided, including mammalian mesenchymal stem cells (MSCs) characterized by the lack of CD54 (CD54?) or low cell-surface CD54 (CD54low). Methods of in vitro cell differentiation and methods of treatment using said isolated populations are also provided.
    Type: Application
    Filed: July 19, 2018
    Publication date: November 15, 2018
    Inventor: Basil M. HANTASH
  • METHOD FOR PRODUCING PANCREATIC BUD CELLS
    Publication number: 20180327719
    Abstract: Provided is a method for generating pancreatic bud cells, comprising the step of culturing PDX1+/NKX6.1? cells in a medium comprising KGF, EGY, a DMP inhibitor and an Akt inhibitor. The cells may be cultured under adherent culture conditions. PDX1+/NKX6.1? cells may be induced from pluripotent stem cells in vitro. The method of this application makes it possible to generate pancreatic bud cells from pluripotent stem cells.
    Type: Application
    Filed: September 16, 2016
    Publication date: November 15, 2018
    Inventors: Kenji OSAFUNE, Taro TOYODA, Azuma KIMURA
  • METHODS AND PRODUCTS FOR TRANSFECTION
    Publication number: 20180327720
    Abstract: The present invention relates in part to methods for producing tissue-specific cells from patient samples, and to tissue-specific cells produced using these methods. Methods for reprogramming cells using RNA are disclosed. Therapeutics comprising cells produced using these methods are also disclosed.
    Type: Application
    Filed: July 17, 2018
    Publication date: November 15, 2018
    Inventors: Matthew ANGEL, Christopher ROHDE
  • Host Cells with Artificial Endosymbionts
    Publication number: 20180327721
    Abstract: The present invention is directed generally to eukaryotic host cells comprising artificial endosymbionts and methods of introducing artificial endosymbionts into eukaryotic host cells. The invention provides artificial endosymbionts that introduce a phenotype to host cells that is maintained in daughter cells. The invention additionally provides eukaryotic host cells containing magnetotactic bacteria.
    Type: Application
    Filed: July 12, 2018
    Publication date: November 15, 2018
    Applicant: Bell Biosystems, Inc.
    Inventors: Caleb B. Bell, III, Alexey Bazarov
  • Methods for Adeno-Associated Viral Vector Production
    Publication number: 20180327722
    Abstract: The invention relates to an adeno-associated virus (AAV) producer cell comprising nucleic acid sequences encoding: rep/cap gene; helper virus genes; and the DNA genome of the AAV vector particle, wherein the nucleic acid sequences are all integrated together at a single locus within the AAV producer cell genome. The invention also relates to nucleic acid vectors comprising a non-mammalian origin of replication and the ability to hold at least 25 kilobases (kb) of DNA, characterized in that the nucleic acid vectors comprise nucleic acid sequences encoding: rep/cap gene, and helper virus genes. The invention also relates to uses and methods using the nucleic acid vectors in order to produce stable AAV packaging and producer cell lines.
    Type: Application
    Filed: April 18, 2018
    Publication date: November 15, 2018
    Inventor: Conrad VINK
  • Production of Glycosylated Nootkatol in Recombinant Hosts
    Publication number: 20180327723
    Abstract: The invention relates to methods for producing glycosylated nootkatol. In particular, a recombinant host comprising a gene encoding a UDP-glycosyltransferase polypeptide capable of glycosylating nootkatol is disclosed. Glycosylation of nootkatol detoxifies nootkatol, allowing for greater production of (glycosylated-)nootkatol, a precursor of nootkatone, and therefore greater production of nootkatone. The invention also relates to methods of converting non-toxic, glycosylated nootkatol produced by a recombinant host to nootkatol, wherein the nootkatol can subsequently be converted to large quantities of nootkatone to be used in flavorings, perfumes, and/or insect repellents.
    Type: Application
    Filed: November 14, 2016
    Publication date: November 15, 2018
    Inventors: Dayal Saran, Jorgen Hansen, Philippe Prochasson, Patrick Roussel, Laura Occhipinti, Alexei Ouspenski, Sumire Honda Malca
  • METHOD FOR PRODUCING HEAVY CHAIN AMINOCARBOXYLIC ACID
    Publication number: 20180327724
    Abstract: The present invention relates to a method for producing a medium chain aminocarboxylic acid and, more particularly, to a recombinant microorganism in which a fatty aldehyde dehydrogenase gene in ?-oxidative metabolic pathway and a ?-oxidative metabolic pathway-related gene are deleted and a ?-transaminase gene is introduced, and a method for producing a medium chain aminocarboxylic acid by culturing the recombinant microorganism. The recombinant microorganism of the present invention can prevent additional oxidation of fatty aldehyde and ?-oxidative metabolism and also produce a medium chain aminocarboxylic acid, such as 12-aminodecane, as a raw material of nylon 12 from a substrate such as fatty acid with a high yield by introducing an amine group into a terminal thereof.
    Type: Application
    Filed: October 27, 2016
    Publication date: November 15, 2018
    Applicant: KOREA RESEARCH INSTITUTE OF BIOSCIENCE AND BIOTECHNOLOGY
    Inventors: Jung Oh AHN, Hong Weon LEE, Gyu Yeon PARK, Min Jeong JANG, Woo Young JEON
  • TATk-CDKL5 Fusion Proteins, Compositions, Formulations, And Use Thereof
    Publication number: 20180327725
    Abstract: Disclosed herein are compositions and formulations containing a TATk-CDKL5 fusion protein.
    Type: Application
    Filed: April 16, 2018
    Publication date: November 15, 2018
    Inventors: Elisabetta Ciani, Franco Laccone
  • ENHANCED hAT FAMILY TRANSPOSON-MEDIATED GENE TRANSFER AND ASSOCIATED COMPOSITIONS, SYSTEMS, AND METHODS
    Publication number: 20180327726
    Abstract: This disclosure provides various TcBuster transposases and transposons, systems, and methods of use.
    Type: Application
    Filed: June 11, 2018
    Publication date: November 15, 2018
    Inventors: David LARGAESPADA, Branden MORIARITY, Beau WEBBER, Neil OTTO, Sandeep KUMAR, Leah HOGDAL
  • ENGINEERED ENZYME HAVING ACETOACETYL-COA HYDROLASE ACTIVITY, MICROORGANISMS COMPRISING SAME, AND METHODS OF USING SAME
    Publication number: 20180327727
    Abstract: The disclosure provides engineered enzymes that are capable of mediating the conversion of acetoacetyl-CoA to acetoacetate that do not react with the same order of magnitude with acetyl-CoA as they do with acetoacetyl-CoA (e.g., the engineered enzymes have a specific acetoacetyl-CoA hydrolase activity at least 10x higher than its acetyl-CoA hydrolase activity). Additionally, the disclosure provides modified microorganisms that comprise the engineered enzymes disclosed herein and methods of using same.
    Type: Application
    Filed: June 18, 2018
    Publication date: November 15, 2018
    Inventors: Avram Michael Slovic, Iuri Estrada Gouvea, Daniel Johannes Koch, Felipe Galzerani
  • ENZYME VARIANTS WITH IMPROVED ESTER SYNTHASE PROPERTIES
    Publication number: 20180327728
    Abstract: The disclosure relates to enzyme variants with improved ester synthase properties for the production of fatty acid esters. Further contemplated are recombinant host cells that express such variants, cell cultures comprising the recombinant host cells and fatty acid ester compositions produced by such recombinant host cells.
    Type: Application
    Filed: January 18, 2018
    Publication date: November 15, 2018
    Inventors: Andrew M. SHUMAKER, Bernardo M. da Costa, Kevin HOLDEN, Louis G. HOM, Tarah S. BARON, Noah HELMAN
  • NOVEL CELLULASE GENE
    Publication number: 20180327729
    Abstract: An object is to identify endoglucanase and ?-glucosidase genes by isolating genomic DNA containing cellulase genes, which are classified into endoglucanases or ?-glucosidases, from Acremonium cellulolyticus, and sequencing the nucleotide sequences thereof. The inventors intensively compared the amino acid sequences of known endoglucanases and ?-glucosidases with each other to find conserved region of amino acid sequences in Acremonium cellulolyticus, and various primers were designed based on the information. PCR was carried out using the various primers thus designed and genomic DNA or cDNA as a template. As a result, gene fragments of endoglucanases and ?-glucosidases were obtained. Primers were designed based on the gene fragments, and PCR was carried out to amplify nine genes of endoglucanases and ?-glucosidases. The nucleotide sequences thereof were sequenced, and the present invention was completed.
    Type: Application
    Filed: July 18, 2018
    Publication date: November 15, 2018
    Applicant: MEIJI SEIKA PHARMA CO., LTD.
    Inventor: Fumikazu Yokoyama
  • NOVEL RECOMBINANT CLOSTRIDIAL NEUROTOXINS WITH INCREASED DURATION OF EFFECT
    Publication number: 20180327730
    Abstract: This invention relates to novel recombinant clostridial neurotoxins exhibiting increased duration of effect and to methods for the manufacture of such recombinant clostridial neurotoxins. These novel recombinant clostridial neurotoxins comprise (i) at least two random coil domains, or (ii) at least two domains having a polyproline II helix conformation, and the methods comprise the steps of inserting at least two nucleic acid sequences each coding for (i) a random coil domain, or (ii) a domain having a polyproline II helix conformation, into a nucleic acid sequence coding for a parental clostridial neurotoxin and expression of the recombinant nucleic acid sequence comprising (i) the random coil domain-coding sequences, or (ii) the sequences encoding the domains having a polyproline II helix conformation in a host cell.
    Type: Application
    Filed: January 19, 2017
    Publication date: November 15, 2018
    Inventors: Fred Hofmann, Marcel Jurk, Manuela López De La Paz, Daniel Scheps, Jürgen Frevert
  • METHOD OF PRODUCING COLLAGENASE
    Publication number: 20180327731
    Abstract: The present invention relates to the fields of collagenase production and collagenase products, and particularly to improving the reproducibility, purity, and stability of collagenase I and collagenase II compositions, where the compositions are pure to at least 95% by area as measured by reverse phase high pressure liquid chromatography (RP-HPLC) and essentially free of neutral protease.
    Type: Application
    Filed: March 28, 2018
    Publication date: November 15, 2018
    Inventors: Christine A. Sheaffer, Michael Berbaum, John Hanna, Jason Dziadosz, Daniel Shanafelt
  • POLYPEPTIDES HAVING PROTEASE ACTIVITY AND POLYNUCLEOTIDES ENCODING SAME
    Publication number: 20180327732
    Abstract: The present invention relates to isolated polypeptides having protease activity, and polynucleotides encoding the polypeptides. The invention also relates to nucleic acid constructs, vectors, and host cells comprising the polynucleotides as well as methods of producing and using the polypeptides.
    Type: Application
    Filed: November 24, 2016
    Publication date: November 15, 2018
    Applicant: NOVOZYMES A/S
    Inventors: Ye Liu, Xianzhi Jiang, Lan Tang, Astrid Benie, Henrik Frisner
  • MONOTERPENE-PRODUCING GENETICALLY MODIFIED HOST CELLS AND METHODS OF USE OF SAME
    Publication number: 20180327733
    Abstract: The present disclosure provides a genetically modified host cell capable of producing linalool (or 3,7-dimethylocta-1,6-dien-3-ol).
    Type: Application
    Filed: December 9, 2016
    Publication date: November 15, 2018
    Inventors: Charles M. Denby, Jay D. Keasling, James Kirby, Rachel Alexandra Li
  • ENGINEERED PRIMATE CYSTINE/CYSTEINE DEGRADING ENZYMES FOR THERAPEUTIC USES
    Publication number: 20180327734
    Abstract: Methods and compositions related to the engineering of a protein with L-cyst(e)ine degrading enzyme activity are described. For example, disclosed are modified cystathionine-?-lyases comprising one or more amino acid substitutions and capable of degrading L-cyst(e)ine. Furthermore, compositions and methods are provided for the treatment of cancer and cystinuria using the disclosed modified enzymes or nucleic acids encoding said enzymes.
    Type: Application
    Filed: May 11, 2018
    Publication date: November 15, 2018
    Applicant: Board of Regents, The University of Texas System
    Inventors: George Georgiou, Everett Stone, Shira Cramer
  • Production of high purity chondroitinase ABC
    Publication number: 20180327735
    Abstract: The present invention provides a method for purifying Chondroitinase ABC (ChABC). The present method includes using a heparin-immobilized affinity chromatography column, and through chromatography method obtaining a purified ChABC from a matrix containing said ChABC. The present method is capable of obtaining ChABC in high purity with the advantages of simplicity in preparation and high yield.
    Type: Application
    Filed: July 30, 2018
    Publication date: November 15, 2018
    Inventors: Bing Lou WONG, Wai Hong CHUNG
  • PEPTIDES DERIVED FROM LENGSIN (LGSN), COMPLEXES COMPRISING SUCH PEPTIDES BOUND TO MHC MOLECULES
    Publication number: 20180327736
    Abstract: The present invention relates to novel peptides derived from Lengsin (LGSN), complexes comprising such peptides bound to recombinant MHC molecules, and cells presenting said peptide in complex with MHC molecules. Also provided by the present invention are binding moieties that bind to the peptides and/or complexes of the invention. Such moieties are useful for the development of immunotherapeutic reagents for the treatment of diseases such as cancer.
    Type: Application
    Filed: November 23, 2016
    Publication date: November 15, 2018
    Inventors: Alex POWLESLAND, Maurits KLEIJNEN
  • APPARATUS AND METHOD FOR DEHYDRATING BIOLOGICAL MATERIALS WITH FREEZING AND MICROWAVING
    Publication number: 20180327737
    Abstract: An apparatus and method for dehydrating biological materials, such as vaccines and microorganism cultures, in which the materials are dehydrated in an evacuated container which is in a microwave waveguide that is open to the atmosphere. The apparatus comprises means for freezing the container of biological material, a microwave generator, a waveguide, means for introducing the container into the waveguide, means for applying a vacuum to the container and means for removing the dehydrated material from the waveguide. In the method of the invention, the container of biological material is put in a microwave waveguide open to the atmosphere, a vacuum is applied to the container, the material is frozen and is radiated to dehydrate it. The dehydrated material is then removed from the waveguide.
    Type: Application
    Filed: July 12, 2018
    Publication date: November 15, 2018
    Inventors: Timothy D. Durance, Jun Fu, Parastoo Yaghmaee, Robert L. Pike
  • STABILIZED REAGENTS FOR GENOME MODIFICATION
    Publication number: 20180327738
    Abstract: The present disclosure generally relates to compositions and methods for genetic modification of cells. In particular, the disclosure relates to stabilized reagents for genome alteration (e.g., site specific nucleases), as well as stabilized reagents for non-genetic code altering modification (e.g., DNA methylation). Stabilization methods include storage of reagents at suitable temperatures, conversion to dry forms, and chemical modifications.
    Type: Application
    Filed: November 18, 2016
    Publication date: November 15, 2018
    Inventors: Namritha RAVINDER, Robert Jason POTTER, Jason CARTE
  • De Novo Design of Allosteric Proteins
    Publication number: 20180327739
    Abstract: Methods and compositions for making and isolating allosteric DNA binding proteins that bind to one or more allosteric effectors to induce a conformation change in the proteins are provided.
    Type: Application
    Filed: July 19, 2018
    Publication date: November 15, 2018
    Inventors: Srivatsan Raman, Noah D. Taylor, George M. Church
  • HIGH-THROUGHPUT CRISPR-BASED LIBRARY SCREENING
    Publication number: 20180327740
    Abstract: Provided herein is an improved method for performing CRISPR/Cas based screening that is not dependent on viral cloning methods.
    Type: Application
    Filed: November 11, 2016
    Publication date: November 15, 2018
    Applicants: Massachusetts Institute of Technology, The Brigham and Women's Hospital, Inc.
    Inventors: David K. Gifford, Nisha Rajagopal, Richard Irving Sherwood
  • NUCLEIC ACID NANOSTRUCTURES WITH CORE MOTIFS
    Publication number: 20180327741
    Abstract: Optimized inhibitory nucleic acids are provided. The nucleic acids have sequences which include an optimal inhibitory motif, such as a GGG. Related methods are also described.
    Type: Application
    Filed: January 14, 2016
    Publication date: November 15, 2018
    Applicant: Exicure, Inc.
    Inventors: Weston DANIEL, Scott MIX
  • Targeted Self-Assembly of Functionalized Carbon Nanotubes on Tumors
    Publication number: 20180327742
    Abstract: Provided herein is the two component self-assembly single wall nanotube system and the single wall nanotube construct that is the second component.
    Type: Application
    Filed: May 22, 2018
    Publication date: November 15, 2018
    Inventors: David A. Scheinberg, Michael R. McDevitt, Carlos H. Villa, J. Justin Mulvey
  • Antisense Polynucleotides to Induce Exon Skipping and Method of Treating Dystrophies
    Publication number: 20180327743
    Abstract: Antisense polynucleotides and their use in pharmaceutical compositions to induce exon skipping in targeted exons of the gamma sarcoglycan gene are provided, along with methods of preventing or treating dystrophic diseases such as Limb-Girdle Muscular Dystrophy.
    Type: Application
    Filed: July 20, 2018
    Publication date: November 15, 2018
    Inventor: Elizabeth McNally
  • iRNA AGENTS WITH BIOCLEAVABLE TETHERS
    Publication number: 20180327744
    Abstract: The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more linking groups, in which at least one of the linking groups is a cleavable linking group. The tether in turn can be connected to a selected moiety, e.g., a ligand, e.g., a targeting or delivery moiety, or a moiety which alters a physical property. The cleavable linking group is one which is sufficiently stable outside the cell such that it allows targeting of a therapeutically beneficial amount of an iRNA agent (e.g., a single stranded or double stranded iRNA agent), coupled by way of the cleavable linking group to a targeting agent—to targets cells, but which upon entry into a target cell is cleaved to release the iRNA agent from the targeting agent.
    Type: Application
    Filed: July 23, 2018
    Publication date: November 15, 2018
    Inventors: Muthiah MANOHARAN, Kallanthottathil G. RAJEEV
  • METHODS OF CONTROLLING CELL FATE AND CONSEQUENCES FOR DISEASE
    Publication number: 20180327745
    Abstract: Provided herein are methods for performing cellular reprogramming that include treatment of somatic cells with an inhibitor of CAF-1, Sumo2, Nutd21, or combinations thereof prior to or during a reprogramming procedure. Such inhibitors can improve both the speed and efficiency of cellular reprogramming. Inhibitors of the CAF-1 complex can also be used in the treatment of cancer.
    Type: Application
    Filed: July 24, 2018
    Publication date: November 15, 2018
    Applicant: THE GENERAL HOSPITAL CORPORATION
    Inventors: Konrad HOCHEDLINGER, Sihem CHELOUFI
  • Alpha-AMYLASE-BINDING NUCLEIC ACID MOLECULE AND USE THEREOF
    Publication number: 20180327746
    Abstract: The present invention provides a novel nucleic acid molecule that can be used for detection of ?-amylase. The ?-amylase-binding nucleic acid molecule of the present invention is characterized in that it binds to ?-amylase with a dissociation constant of 17 nM or less, and preferably includes a polynucleotide consisting of any of base sequences of SEQ ID NOs: 1 to 22, for example. According to the nucleic acid molecule of the present invention, it is possible to detect ?-amylase in saliva.
    Type: Application
    Filed: September 12, 2016
    Publication date: November 15, 2018
    Applicants: NEC Solution Innovators, Ltd., Gunma University
    Inventors: Hirotaka MINAGAWA, Katsunori HORII, Jou AKITOMI, Naoto KANEKO, Ikuo SHIRATORI, Iwao WAGA, Masayasu KUWAHARA
  • EXOGENOUS CONTROL OF MAMMALIAN GENE EXPRESSION THROUGH APTAMER-MEDIATED MODULATION OF POLYADENYLATION
    Publication number: 20180327747
    Abstract: Embodiments of the disclosure concern the use of expression constructs in which at least one polyA signal is embedded upstream of an expressible transcript, such as within a 5? UTR for the transcript, for example. In certain embodiments, the polyA signal is comprised within a ligand-binding aptamer, and the binding of the ligand to the aptamer, or lack thereof, dictates the outcome for the expressible transcript. In specific embodiments, absence of the ligand causes the expressed transcript having a polyA in its 5? UTR to be expressed but then degraded, whereas presence of the ligand causes inhibition of degradation upon expression of the expressible transcript. More than one ligand-binding aptamer may be present on the same expression construct.
    Type: Application
    Filed: November 11, 2016
    Publication date: November 15, 2018
    Inventors: Laising Yen, Liming Luo, Pei-Wen Chao
  • RNA INTERFERENCE IN SKIN INDICATIONS
    Publication number: 20180327748
    Abstract: The present invention relates to RNAi constructs with improved tissue and cellular uptake characteristics and methods of use of these compounds in dermal applications.
    Type: Application
    Filed: February 26, 2018
    Publication date: November 15, 2018
    Applicant: RXi Pharmaceuticals Corporation
    Inventors: Anastasia Khvorova, William Salomon, Joanne Kamens, Dmitry Samarsky, Tod M. Woolf, Pamela A. Pavco, James Cardia
  • ANTISENSE COMPOSITION AND METHOD FOR TREATING MUSCLE ATROPHY
    Publication number: 20180327749
    Abstract: A method and compound for treating skeletal muscle mass deficiency in a human subject are disclosed. The composition is an oligomer of morpholino subunits and phosphorus-containing intersubunit linkages joining a morpholino nitrogen of one subunit to a 5? exocyclic carbon of an adjacent subunit, contains between 10-40 nucleotide bases, has a base sequence effective to hybridize to an expression-sensitive region of processed or preprocessed human myostatin RNA transcript, identified, in its processed form, by SEQ ID NO:6, and is capable of uptake by target muscle cells in the subject. In practicing the method, the compound is administered in an amount and at a dosage schedule to produce an overall reduction in the level of serum myostatin measured in the patient, and preferably to bring the myostatin level within the a range determined for normal, healthy individuals.
    Type: Application
    Filed: May 22, 2018
    Publication date: November 15, 2018
    Inventors: Patrick L. Iversen, Dwight D. Weller, Alan P. Timmins
  • Methods and Compositions for Reducing Immunosupression by Tumor Cells
    Publication number: 20180327750
    Abstract: The present disclosure provides, in part, methods of discovering immunotherapy targets in vivo, therapeutic compositions (e.g., shRNA, immunoresponsive cells expressing shRNA and/or a chimeric antigen receptors (CAR)), and methods of use thereof.
    Type: Application
    Filed: April 3, 2018
    Publication date: November 15, 2018
    Applicants: Dana-Farber Cancer Institute, Inc., The General Hospital Corporation d/b/a Massachusetts General Hospital
    Inventors: Kai W. Wucherpfennig, Glenn Dranoff, Penghui Zhou, Donald Shaffer, Nir Hacohen, Harvey I. Cantor, Diana Alvarez Arias
  • siRNA COMPOSITIONS THAT SPECIFICALLY DOWNREGULATE EXPRESSION OF A VARIANT OF THE PNPLA3 GENE AND METHODS OF USE THEREOF FOR TREATING A CHRONIC LIVER DISEASE OR ALCOHOLIC LIVER DISEASE (ALD)
    Publication number: 20180327751
    Abstract: The invention provides siRNA compositions that specifically downregulates expression of a variant of the PNPLA3 gene and methods of use thereof for treating a chronic liver disease or alcoholic liver disease (ALD).
    Type: Application
    Filed: July 3, 2018
    Publication date: November 15, 2018
    Inventors: Wanqing Liu, Yoon Yeo
  • METHODS AND COMPOSITIONS FOR MODULATING AAV INFECTION
    Publication number: 20180327752
    Abstract: Compositions and methods are provided for modulating adeno-associated virus (AAV) infection. For example, compositions and methods are provided for enhancing permissiveness of a target cell to AAV infection (e.g., by increasing levels of AAVR (KIAA0319L) in the cell), for reducing permissiveness of a target cell to AAV infection (e.g., by reducing levels of AAVR in the cell), and for nucleic acid delivery (e.g., by (i) increasing permissiveness of a target cell to AAV infection, e.g., by increasing the amount of AAVR in the cell; and (ii) contacting the target cell with an AAV particle that includes a nucleic acid of interest). Also provided are screening methods and kits for practicing the methods of the disclosure.
    Type: Application
    Filed: November 9, 2016
    Publication date: November 15, 2018
    Inventors: Sirika Pillay, Jan Carette, Michael Stewart Chapman, Nancy Meyer, Andreas Puschnik, Omar Davulcu
  • MEANS AND METHODS FOR REDUCING TUMORIGENICITY OF CANCER STEM CELLS
    Publication number: 20180327753
    Abstract: Provided are RNA inhibitory molecules, particularly siRNA molecules silencing the expression of Voltage-Dependent Ion Chanel-1 (VDAC1) for the induction of cancer stem cell differentiation and reduction of TAMs abundance and tumor angiogenesis, thereby preventing tumor invention and recurrence.
    Type: Application
    Filed: November 10, 2016
    Publication date: November 15, 2018
    Applicants: B. G. NEGEV TECHNOLOGIES AND APPLICATIONS LTD., AT BEN-GURION UNIVERSITY, THE NATIONAL INSTITUTE FOR BIOTECHNOLOGY IN THE NEGEV LTD.
    Inventor: Varda SHOSHAN-BARMATZ
  • Modulation of CCR10 Signals for Treatment of Skin and Intestinal Inflammatory Diseases and Infection
    Publication number: 20180327754
    Abstract: The invention provides compositions and methods for targeting CCR10 and/or the CCR10/ligand axis to modulate the immune response in a subject.
    Type: Application
    Filed: July 20, 2018
    Publication date: November 15, 2018
    Inventors: Na Xiong, Mingcan Xia
  • TREATMENT OF AGE-RELATED MACULAR DEGENERATION USING RNA COMPLEXES THAT TARGET MYD88 OR TLR3
    Publication number: 20180327755
    Abstract: In certain aspects, provided herein are RNA complexes that inhibit Myeloid differentiation primary response gene 88 (MyD88) and/or Toll-like receptor 3 (TLR3) and are useful in the treatment of age-related macular degeneration (AMD). In certain aspects, provided herein are pharmaceutical compositions comprising such RNA complexes and methods of using such RNA complexes and pharmaceutical compositions.
    Type: Application
    Filed: July 23, 2018
    Publication date: November 15, 2018
    Inventors: Dong-ki Lee, Sun Woo Hong, Isu Hong, Jihye Hwang
  • CRISPR-CAS COMPONENT SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION
    Publication number: 20180327756
    Abstract: The invention provides for systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for selecting specific cells by introducing precise mutations utilizing the CRISPR/Cas system.
    Type: Application
    Filed: August 5, 2016
    Publication date: November 15, 2018
    Inventors: Feng Zhang, Le Cong, David Benjamin Turitz Cox, Patrick Hsu, Shuailiang Lin, Fei Ran, Randall Jeffrey Platt, Neville Espi Sanjana