Patents Issued in May 17, 2018
  • Publication number: 20180135012
    Abstract: Compositions comprising synthetic membrane-receiver complexes, methods of generating synthetic membrane-receiver complexes, and methods of treating or preventing diseases, disorders or conditions therewith.
    Type: Application
    Filed: May 13, 2016
    Publication date: May 17, 2018
    Inventors: Jordi Mata-Fink, Avak Kahvejian
  • Publication number: 20180135013
    Abstract: The invention provides a chimeric receptor comprising NKG2D, DAP10 and CD3 zeta. Also disclosed is a composition comprising this chimeric receptor and methods for making and using it to enhance the cytotoxicity and antitumor capacity of NK cells. The invention also encompasses methods for use of NKG2D-DAP10-CD3 zeta polypeptides, vectors and cells in methods for treating cancer and other proliferative disorders, as well as infectious diseases.
    Type: Application
    Filed: December 28, 2017
    Publication date: May 17, 2018
    Applicants: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC., NATIONAL UNIVERSITY OF SINGAPORE
    Inventors: Dario Campana, Yu-Hsiang Chang
  • Publication number: 20180135014
    Abstract: The invention provides a chimeric receptor comprising NKG2D, DAP10 and CD3 zeta. Also disclosed is a composition comprising this chimeric receptor and methods for making and using it to enhance the cytotoxicity and antitumor capacity of NK cells. The invention also encompasses methods for use of NKG2D-DAP10-CD3 zeta polypeptides, vectors and cells in methods for treating cancer and other proliferative disorders, as well as infectious diseases.
    Type: Application
    Filed: December 28, 2017
    Publication date: May 17, 2018
    Applicants: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC., NATIONAL UNIVERSITY OF SINGAPORE
    Inventors: Dario Campana, Yu-Hsiang Chang
  • Publication number: 20180135015
    Abstract: The invention provides a chimeric receptor comprising NKG2D, DAP10 and CD3 zeta. Also disclosed is a composition comprising this chimeric receptor and methods for making and using it to enhance the cytotoxicity and antitumor capacity of NK cells. The invention also encompasses methods for use of NKG2D-DAP10-CD3 zeta polypeptides, vectors and cells in methods for treating cancer and other proliferative disorders, as well as infectious diseases.
    Type: Application
    Filed: December 28, 2017
    Publication date: May 17, 2018
    Applicants: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC, NATIONAL UNIVERSITY OF SINGAPORE
    Inventors: Dario Campana, Yu-Hsiang Chang
  • Publication number: 20180135016
    Abstract: The invention provides a chimeric receptor comprising NKG2D, DAP10 and CD3 zeta. Also disclosed is a composition comprising this chimeric receptor and methods for making and using it to enhance the cytotoxicity and antitumor capacity of NK cells. The invention also encompasses methods for use of NKG2D-DAP10-CD3 zeta polypeptides, vectors and cells in methods for treating cancer and other proliferative disorders, as well as infectious diseases.
    Type: Application
    Filed: December 28, 2017
    Publication date: May 17, 2018
    Applicants: ST. JUDE CHILDREN'S RESEARCH HOSPITAL, INC., NATIONAL UNIVERSITY OF SINGAPORE
    Inventors: Dario Campana, Yu-Hsiang Chang
  • Publication number: 20180135017
    Abstract: The present disclosure describes a method and composition for enhancing the survival of hematopoietic stem cells, preferably CD34+ derived from human umbilical cord or peripheral blood, in hypoxic and serum-deprived conditions by cultivating the cells in medium containing lysophosphatidic acid, preferably further comprising a gel, namely a biomimetic gel.
    Type: Application
    Filed: January 12, 2018
    Publication date: May 17, 2018
    Applicant: Stemlab, S.A.
    Inventors: Lino da Silva Ferreira, Isabel Maria Fidalgo dos Santos Silva Carvalho
  • Publication number: 20180135018
    Abstract: Aspects of the present invention include methods and compositions related to the production and use of embryonic progenitor cell types useful in the generation of cartilage, bone, choroid plexus, tendon, lipasin-secreting adipocytes, and other differentiated cell types useful in research and therapy.
    Type: Application
    Filed: June 12, 2017
    Publication date: May 17, 2018
    Inventors: Michael West, Karen Chapman, Hal Sternberg, Francois Binette
  • Publication number: 20180135019
    Abstract: The present disclosure relates to methods and products for enriching and isolating stromal stem cells. Certain embodiments of the present disclosure provide a method of enriching for immature, uncommitted stromal stem cells from a population of cells comprising stromal stem cells, the method comprising enriching stromal stem cells from the population of cells using a HSC70 binding agent.
    Type: Application
    Filed: June 1, 2016
    Publication date: May 17, 2018
    Inventors: Andrew Zannettino, Stan Gronthos, Stephen Fitter
  • Publication number: 20180135020
    Abstract: The described invention provides a method of functionally reprogramming adult cells to an immature cell type that expresses one or more embryonic biomarkers with a platelet rich fraction comprising platelet-like cells from umbilical cord blood or peripheral blood, and expanding the immature cell type in vitro under culture conditions to generate an insulin-producing cell population that expresses human beta-cell specific transcription factors and is functionally equivalent to human pancreatic beta-cells. It further provides a pharmaceutical composition comprising a cell product containing a therapeutic amount of an insulin-producing cell population, wherein the insulin-producing cell population expresses human beta-cell specific transcription factors and is functionally equivalent to human pancreatic beta-cells, and a method for treating a recipient subject suffering from a disease characterized by hyperglycemia with the pharmaceutical composition.
    Type: Application
    Filed: August 28, 2017
    Publication date: May 17, 2018
    Inventor: Yong Zhao
  • Publication number: 20180135021
    Abstract: A novel method of inducing or producing pancreatic beta cells from human induced pluripotent stem cells at an unprecedented efficiency and functionality. The core of the invention is the use of experimentally discovered mRNAs at multiple critical differentiation decision points along a pluripotent to mesendoderm to endoderm to pancreatic endocrine cells to pancreatic beta cells pathway in a previously unknown manner.
    Type: Application
    Filed: November 16, 2017
    Publication date: May 17, 2018
    Inventors: Jiwu WANG, Yuhui NI, Yuanyuan ZHAO
  • Publication number: 20180135022
    Abstract: According to the present invention, there is provided a method for producing artificial trophoblasts derived from human cells, that includes the steps of: adhesion culturing human pluripotent stem cells in a culture medium containing a BMP signal transduction activator, and during the culturing bringing the cells under culturing into contact with at least one selected from the group consisting of a ? aminobutyric acid B receptor activator, a peroxisome proliferator-activated receptor ? activator, a retinoid X receptor activator, and a retinoic acid receptor activator, thereby obtaining a culture containing trophoblasts differentiated from the human pluripotent stem cells, and so on.
    Type: Application
    Filed: May 16, 2016
    Publication date: May 17, 2018
    Inventors: Kouji ASANO, Koichi SAITO, Tomo MAHARA
  • Publication number: 20180135023
    Abstract: A novel method of inducing or producing hepatocytes from human induced pluripotent stem cells at an unprecedented efficiency and functionality. The core of the invention is the use of experimentally discovered mRNAs at multiple critical differentiation decision points along a pluripotent to mesendoderm to endoderm to hepatocytes pathway in a previously unknown manner.
    Type: Application
    Filed: November 16, 2017
    Publication date: May 17, 2018
    Inventors: Jiwu WANG, Yuhui NI, Yuanyuan ZHAO
  • Publication number: 20180135024
    Abstract: Methods for isolating and optionally purifying AAV particles grown in cell cultures using a combination of lyotropic salts, removal of insoluble producer cell debris and DNA, and optional fractionation by hydrophobic interaction chromatography are described.
    Type: Application
    Filed: November 14, 2016
    Publication date: May 17, 2018
    Applicant: St. Jude Children's Research Hospital
    Inventors: David Joseph McNally, Bryan Andrew Piras, Michael Martin Meagher
  • Publication number: 20180135025
    Abstract: The present invention relates to a retroviral system for the transfer of non-viral RNA into target cells and more particularly a retroviral particle capable of delivering multiple RNAs. More particularly, it relates to retroviral particles comprising a protein derived from the Gag polyprotein an envelope protein, optionally an integrase and at least two encapsidated non-viral RNAs, the encapsidated non-viral RNAs each comprising an RNA sequence of interest linked to an encapsidation sequence, each encapsidation sequence being recognised by a binding domain introduced into the protein derived from the Gag polyprotein and/or into the integrase.
    Type: Application
    Filed: May 13, 2016
    Publication date: May 17, 2018
    Inventors: Pascale BOUILLE, Jean-Christophe PAGES, Régis GAYON
  • Publication number: 20180135026
    Abstract: The present invention relates to the development and manufacturing of viral vaccines. In particular, the invention relates to the field of industrial production of viral vectors and vaccines, more in particular to the use of avian embryonic stem cells, preferably the EBx® cell line derived from duck embryonic stem cells, for the production of viral vectors and viruses. The invention is particularly useful for the industrial production of viral vaccines to prevent viral infection of humans and animals.
    Type: Application
    Filed: October 17, 2017
    Publication date: May 17, 2018
    Applicant: Valneva
    Inventors: Fabienne Guehenneux, Karine Moreau, Magali Esnault, Majid Mehtali
  • Publication number: 20180135027
    Abstract: Nucleic acids encoding Parvoviral Rep proteins with a mutated nuclear localization signal (NLS) are provided. Also provided is a nucleic acid comprising a nucleotide sequence encoding a Parvoviral Rep protein with a mutated zinc finger domain and a nucleic acid comprising a nucleotide sequence encoding a Parvoviral Rep protein comprising an amino acid mutation at position 43, 57, 79, 97, 120, 179, 305, 484, 493 or 571 with reference to SEQ ID NO: 2. Nucleic acid constructs and cells, such as insect cells, comprising the nucleic acids are provided as well as a method for producing a recombinant Parvoviral virion using the nucleic acids.
    Type: Application
    Filed: January 5, 2018
    Publication date: May 17, 2018
    Applicant: uniQure IP B.V.
    Inventors: Yvet NOORDMAN, Jacek LUBELSKI, Andrew Christian BAKKER
  • Publication number: 20180135028
    Abstract: The disclosure relates to variant carboxylic acid reductase (CAR) enzymes for the improved production of fatty alcohols in recombinant host cells.
    Type: Application
    Filed: September 5, 2017
    Publication date: May 17, 2018
    Inventors: Derek L. GREENFIELD, Elizabeth J. CLARKE, Eli S. GROBAN, Vikranth Arlagadda, Sungwon LEE, Xuezhi LI, Zhihao Hu, Baolong ZHU
  • Publication number: 20180135029
    Abstract: A method is proposed for producing flavonoids, comprising the steps: (a) providing of a transgenic microorganism, containing (i) a first nucleic acid section (A), comprising or consisting of a gene coding for a CYP450 oxidase, (ii) a second nucleic acid section (B), comprising or consisting of a gene coding for a plant O-methyltransferase, and (b) adding of one or more flavanones to the transgenic microorganism, (c) the conversion of the substrate flavanones by the transgenic microorganism to the corresponding flavonoids, and optionally (d) isolating and purifying of the final products.
    Type: Application
    Filed: September 27, 2015
    Publication date: May 17, 2018
    Inventors: Ludger Wessjohann, Anne-Kathrin Bauer, Jakob Ley, Torsten Geissier, Katrin Geissler
  • Publication number: 20180135030
    Abstract: Non-viral delivery systems comprising engineered hOTC DNA and RNA sequences are provided which when delivered to a subject in need thereof are useful for treating hyperammonemia, ornithine transcarbamylase deficiency and symptoms associated therewith. Also provided are methods of using hOTC for treatment of liver fibrosis and/or cirrhosis in OTCD patients by administering hOTC.
    Type: Application
    Filed: January 3, 2018
    Publication date: May 17, 2018
    Inventors: Lili Wang, James M. Wilson
  • Publication number: 20180135031
    Abstract: The present invention relates to a microorganism having improved L-lysine-producing ability and an L-lysine-producing method using the same. More specifically, the present invention relates to a microorganism of the genus of Corynebacterium, in which acetate kinase activity is further enhanced over inherent activity, and an L-lysine-producing method using the same.
    Type: Application
    Filed: April 7, 2015
    Publication date: May 17, 2018
    Inventors: Hyun Won Bae, Jun Ok Moon, Sung Ki Song, Hyung Joon Kim, Hyo Jin Kim, Sang Jo Lim
  • Publication number: 20180135032
    Abstract: The present invention refers to hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB). The invention further refers to corresponding nucleic acids producing these variants, to a gene transfer system for stably introducing nucleic acid(s) into the DNA of a cell by using these hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB) and to transposons used in the inventive gene transfer system, comprising a nucleic acid sequence with flanking repeats (IRs and/or RSDs). Furthermore, applications of these transposase variants, the transposon, or the gene transfer system are also disclosed such as gene therapy, insertional mutagenesis, gene discovery (including genome mapping), mobilization of genes, library screening, or functional analysis of genomes in vivo and in vitro. Finally, pharmaceutical compositions and kits are also encompassed.
    Type: Application
    Filed: November 3, 2017
    Publication date: May 17, 2018
    Applicant: MAX-DELBRÜCK-CENTRUM FÜR MOLEKULARE MEDIZIN
    Inventors: Zsuzsanna Izsvak, Zoltan Ivics, Lajos Mates, Namitha Manoj, Carmen-Anisia Judis, Andrea Katzer
  • Publication number: 20180135033
    Abstract: Disclosed are DNA polymerases having increased reverse transcriptase efficiency relative to a corresponding, unmodified polymerase. The polymerases are useful in a variety of disclosed primer extension methods. Also disclosed are related compositions, including recombinant nucleic acids, vectors, and host cells, which are useful, e.g., for production of the DNA polymerases.
    Type: Application
    Filed: January 4, 2018
    Publication date: May 17, 2018
    Inventors: Keith Bauer, Thomas W. Myers, Shawn Suko
  • Publication number: 20180135034
    Abstract: Disclosed are mutant DNA polymerases having increased 3?-mismatch discrimination relative to a corresponding, unmodified polymerase. The mutant polymerases are useful in a variety of disclosed primer extension methods. Also disclosed are related compositions, including recombinant nucleic acids, vectors, and host cells, which are useful, e.g., for production of the mutant DNA polymerases.
    Type: Application
    Filed: January 4, 2018
    Publication date: May 17, 2018
    Inventors: Keith Bauer, Fred Reichert, Thomas W. Myers
  • Publication number: 20180135035
    Abstract: The present invention relates to isolated polypeptides having xylanase 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: December 8, 2017
    Publication date: May 17, 2018
    Applicant: Novozymes, Inc.
    Inventor: Nikolaj Spodsberg
  • Publication number: 20180135036
    Abstract: The present invention relates to the use of isolated polypeptides having protease activity in animal feed and detergents. It also relates to the use of isolated nucleic acid sequences encoding the proteases in the recombinant production of isolated polypeptides having protease activity and isolated nucleic acid sequences encoding the proteases. The invention also relates to nucleic acid constructs, vectors, and host cells, including plant and animal cells, comprising the nucleic acid sequences, as well as methods for producing and using the proteases, particularly using the proteases in animal feed and detergents.
    Type: Application
    Filed: January 2, 2018
    Publication date: May 17, 2018
    Applicant: Novozymes A/S
    Inventors: Tine Hoff, Robert Piotr Olinski, Carsten Sjoeholm, Peter Rahbek Oestergaard, Katrine Fruegaard Pontoppidan, Astrid Benie, Morten Gjermansen
  • Publication number: 20180135037
    Abstract: Isolated and/or purified polypeptides and nucleic acid sequences encoding polypeptides from Alicyclobacillus acidocaldarius are provided. Further provided are methods for modulating or altering metabolism in a cell using isolated and/or purified polypeptides and nucleic acid sequences from Alicyclobacillus acidocaldarius.
    Type: Application
    Filed: January 9, 2018
    Publication date: May 17, 2018
    Inventors: Vicki S. Thompson, William A. Apel, Jeffrey A. Lacey, Brady D. Lee, David W. Reed, Francisco F. Roberto, David N. Thompson
  • Publication number: 20180135038
    Abstract: Disclosed is an enzymatic composition including:—at least one enzyme, termed plasminogenase, for converting, into plasmin, plasminogen from a blood plasma medium including plasminogen;—a solid support that is insoluble in aqueous solution, the solid support having dimensions suitable for being able to be retained on a filter having a cut-off threshold of less than or equal to 0.22 ?m. The plasminogenase is bound to the solid support and remains bound to this support on contact with a blood plasma medium. The composition is in the dry state. Also disclosed is a process for preparing such an enzymatic composition, to the use thereof and to a device (20) for preparing a blood plasma medium ex vivo rich in sterile plasmin and free of enzymatic composition.
    Type: Application
    Filed: April 14, 2016
    Publication date: May 17, 2018
    Inventors: Gerard REBOUL, Carine SAUX, Francoise OUARNE
  • Publication number: 20180135039
    Abstract: The present invention relates to improved yeast transformation of yeast cells and yeast cell libraries transformed thereby. More specifically, the present invention relates to the transformation of yeast by electroporation.
    Type: Application
    Filed: November 15, 2017
    Publication date: May 17, 2018
    Inventors: Sebastian BUNK, Dominik MAURER, Felix UNVERDORBEN
  • Publication number: 20180135040
    Abstract: Methods for separating and identifying nucleic acids utilize carbon coated magnetic beads. The method teaches that multivalent cations promote binding of single stranded nucleic acids to the beads and that the single stranded nucleic acids can be released with the addition of chelating agents that bind the multivalent cations such as EDTA. The method further teaches that fragile single stranded nucleic acids, such as RNA, can be stored on the surface of the beads. Lastly, the method also teaches that by iteratively adding complimentary DNA oligos, single stranded nucleic acids can be quantified or individually isolated using the carbon coated magnetic beads.
    Type: Application
    Filed: December 29, 2017
    Publication date: May 17, 2018
    Inventors: Kevin Hagedorn, Saravana Murthy
  • Publication number: 20180135041
    Abstract: Described is a process for producing a mutant bacterium which exhibits improved survival and/or growth under a selected growth condition, the process comprising the steps of: (a) generating a pool of mutant bacteria by transposon mutagenesis with an activating transposon (TnA), wherein the TnA comprises a promoter capable of increasing transcription of a gene at or near its insertion site; (b) growing bacteria from the mutant pool under the selected growth condition and under one or more reference conditions to produce two or more test cultures; and (c) comparing the distribution of TnA insertions between test cultures to identify a first class of genes which are disadvantageous for growth and/or survival under the selected growth condition and a second class of genes which are advantageous for growth and/or survival under the selected growth condition.
    Type: Application
    Filed: November 20, 2017
    Publication date: May 17, 2018
    Inventors: David Hugh Williams, Arthur Keith Turner, John Richard Wain
  • Publication number: 20180135042
    Abstract: Various approaches for generating read-sets from nucleic acid molecules and segments and phasing are disclosed. Nucleic acids are assembled into complexes using binding moieties and exposed nucleic acid ends are tagged with nucleic acid tags. Read-sets can be generated from tagged nucleic acid molecules and segments. Physical linkage relationships between nucleic acid molecules and segments can be examined using the nucleic acid tags. Various approaches to generating read-sets and phasing are presented.
    Type: Application
    Filed: April 4, 2016
    Publication date: May 17, 2018
    Inventor: Daniel S. ROKHSAR
  • Publication number: 20180135043
    Abstract: The invention provides compositions comprising nucleic acid complexes for use in screening compounds based on their ability to modulate binding interactions, wherein the compounds are barcoded.
    Type: Application
    Filed: June 2, 2016
    Publication date: May 17, 2018
    Applicant: Children's Medical Center Corporation
    Inventors: Wesley Philip Wong, Clinton H. Hansen
  • Publication number: 20180135044
    Abstract: The present disclosure involves ctDNA assays that interrogate many regions from a single sample with high precision and accuracy, while evaluating multiple forms of cancer-related genomic alterations including sequence mutations and structural alterations. The disclosure provides simplified yet robust methods that achieve high sensitivity and specificity by analyzing cancer genes using a limited pool of non-unique barcodes in combination with endogenous barcodes. Samples are captured and sequenced using high coverage next-generation sequencing to allow tumor-specific somatic mutations, amplifications, and translocations to be identified.
    Type: Application
    Filed: November 14, 2017
    Publication date: May 17, 2018
    Inventors: Mark Sausen, Victor Velculescu, Luis Diaz
  • Publication number: 20180135045
    Abstract: Compositions and methods for isolating new variants of known gene sequences are provided. The methods find use in identifying variants, particularly homologs, in complex mixtures. Compositions comprise hybridization baits that hybridize to gene families of interest, particularly agricultural interest, in order to selectively enrich the polynucleotides of interest from complex mixtures. Bait sequences may be specific for a number of genes from distinct gene families of interest and may be designed to cover each gene of interest by at least 2-fold. Thus methods disclosed herein are drawn to an oligonucleotide hybridization gene capture approach for identification of new genes of interest from environmental samples.
    Type: Application
    Filed: January 4, 2018
    Publication date: May 17, 2018
    Applicant: AgBiome, Inc.
    Inventors: Vadim Beilinson, Janice Jones, Jessica Parks, Rebecca E. Thayer, Daniel J. Tomso, Scott Joseph Uknes, Sandy Volrath, Eric Russell Ward
  • Publication number: 20180135046
    Abstract: A method of preparing a library of small interfering RNA (siRNA) expression systems for producing siRNA for silencing of target genes by inducing degradation of target gene RNA expression products includes: (i) isolating RNA of one or more target genes from a cell population; (ii) generating RNA fragments from the isolated RNA; (iii) converting the RNA fragments into dsDNA fragments; and (iv) cloning the dsDNA fragments into vectors for forming cloned vectors, each vector including one or more promoters and at least one restriction enzyme site capable of accepting the insertion of at least one dsDNA fragment such that siRNA can be produced. Methods for producing siRNA from the siRNA expression system and methods of identifying a functional target gene for treatment by using the siRNA produced from the siRNA expression system and for identifying RNAi therapeutics are also provided.
    Type: Application
    Filed: November 15, 2016
    Publication date: May 17, 2018
    Inventors: Linfeng Huang, Yutian Ren, Hung-chi Cheung, Guneet Kaur
  • Publication number: 20180135047
    Abstract: The present invention provides an oligodeoxy nucleotide for preparing drugs for inhibiting tumor growth and an application thereof. The core sequence of the oligodeoxy nucleotide is RGGGAHTTYCS, wherein R is G or A; H is A or C or T; Y is C or T; S is C or G. The oligodeoxy nucleotide above further comprises an antisense strand and a modified type thereof. The oligodeoxy nucleotide according to the present invention plays a role of inhibiting the tumor growth in vitro and vivo, and is expected to be used for preparing drugs for inhibiting tumor growth, with high specificity and high inhibition ratio.
    Type: Application
    Filed: June 25, 2015
    Publication date: May 17, 2018
    Inventors: Xuegen Wang, Qing Ye, Yijun Sun
  • Publication number: 20180135048
    Abstract: Methods and compositions are provided which employ a silencing element that, when ingested by a plant insect pest, such as a Coleopteran plant pest or a Diabrotica plant pest, decrease the expression of a target sequence in the pest. Disclosed are various target polynucleotides set forth in any one of SEQ ID NOS: 1-54 and 81-84 disclosed herein, or variants and fragments thereof, or complements thereof, wherein a decrease in expression of one or more of the sequences in the target pest controls the pest (i.e., has insecticidal activity). Plants, plant parts, bacteria and other host cells comprising the silencing elements or an active variant or fragment thereof of the invention are also provided.
    Type: Application
    Filed: February 24, 2016
    Publication date: May 17, 2018
    Inventors: XU HU, Xiping NIU, James Kevin PRESNAIL, Nina RICHTMAN, Joe ZHAO
  • Publication number: 20180135049
    Abstract: The present disclosure provides for methods of treating or preventing a cardiovascular disorder and/or a related pulmonary disorder in a subject. In certain embodiments, a therapeutically effective amount of a polynucleotide inhibitor of Glucose-6-phosphate dehydrogenase (G6PD) or a nucleic acid encoding G6PD is administered.
    Type: Application
    Filed: November 15, 2017
    Publication date: May 17, 2018
    Inventor: Rakhee Gupte
  • Publication number: 20180135050
    Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Alpha-L-Iduronidase (IDUA), in particular, by targeting natural antisense polynucleotides of Alpha-L-Iduronidase (IDUA). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of IDUA.
    Type: Application
    Filed: December 19, 2017
    Publication date: May 17, 2018
    Inventors: Joseph COLLARD, Olga KHORKOVA SHERMAN, Carlos COITO, Gang SHEN
  • Publication number: 20180135051
    Abstract: This invention provides pharmaceutical compositions containing a UNA oligomer targeted to TTR and a pharmaceutically acceptable carrier. The compositions can be used in methods for treating or preventing TTR-related amyloidosis in a primate. The compositions, upon administering a single dose to the primate, can reduce TTR protein in the primate for a period of days to weeks.
    Type: Application
    Filed: December 31, 2017
    Publication date: May 17, 2018
    Inventors: Kiyoshi Tachikawa, Joseph E. Payne, Padmanabh Chivukula
  • Publication number: 20180135052
    Abstract: Provided are aptamers able to bind to ligands associated with cancer cells. The ligands may particularly be associated with brain cancers, such as gliomas. The aptamers may be used therapeutically for the prevention and/or treatment of such cancers. Aptamers may be associated with anti-cancer agents, or with detection moieties. Also provided as pharmaceutical compositions and methods of treatment employing such aptamers.
    Type: Application
    Filed: November 2, 2017
    Publication date: May 17, 2018
    Applicant: University of Central Lancashire
    Inventors: Lisa Shaw, Clare Lawrence, Jane Adler
  • Publication number: 20180135053
    Abstract: A method of stimulating a TLR9-activated immune response or enhancing a TLR9-activated immune response to an antigen is disclosed herein. TLR9 is the cellular receptor for CpG-ODN, and current developed CpG-ODN has low activity to rabbit TLR9. Here, the method of stimulating TLR9-activated immune response by administering an effective amount of immunogenic composition comprising an antigen and a CpG-ODN comprising GACGTT or AACGTT motif was demonstrated to have potent immunostimulatory activity to rabbit TLR9, and capable of boosting a less toxic and potent antibody response in rabbits.
    Type: Application
    Filed: December 29, 2017
    Publication date: May 17, 2018
    Inventors: Tsung-Hsien CHUANG, Chao-Yang LAI
  • Publication number: 20180135054
    Abstract: Modified oligonucleotides comprising modifications at the 2? and/or 3? positions(s) along with methods of making and use, e.g., against HBV are disclosed.
    Type: Application
    Filed: September 14, 2017
    Publication date: May 17, 2018
    Applicant: Alios Biopharma, Inc.
    Inventors: Sergei GRYAZNOV, Leonid Beigelman, Antitsa Dimitrova Stoycheva, Saul Martinez Montero, Jin Hong
  • Publication number: 20180135055
    Abstract: The present invention relates to a composition for cancer cell sensitization containing as an active ingredient a substance inhibiting the expression of an oncogene of a HPV virus, and more specifically, to a composition for radiosensitization which is used for the treatment of cancer induced by HPV infection and contains as an active ingredient oligonucleotide having any one nucleic acid sequence selected from the group consisting of sequence numbers 1 to 10. The oligonucleotide of the present invention inhibits the expressions of E6 and E7 which are HPV virus oncoproteins, thereby inhibiting the proliferation of tumor cells, and enhances the sensitivity of tumor cells to radiation, thereby exhibiting an excellent effect as a sensitizer that can maximize the cell death effect.
    Type: Application
    Filed: January 30, 2018
    Publication date: May 17, 2018
    Inventors: Young Kee Shin, Young Deug Kim, Hun Soon Jung, Deuk Ae Kim, Kyung Tae Ha
  • Publication number: 20180135056
    Abstract: A method of producing nanovesicles comprising an oligonucleotide inhibitor to an oncogene or a proto-oncogene or the gene product thereof, said method comprises a) introducing a DNA sequence encoding an oligonucleotide capable of inhibiting a human oncogenic or proto-oncogenic transcription factor, into a mammalian cell; b) allowing the cell to express said inhibitor oligonucleotide; and c) obtaining nanovesicles containing said inhibitor oligonucleotide from said cell. Nanovesicles produced by the claimed method can be effectively and specifically targeted to e.g. cancer cells to deliver the inhibitor oligonucleotide.
    Type: Application
    Filed: November 28, 2017
    Publication date: May 17, 2018
    Inventors: Jan LÖTVALL, Jonas Andrej NILSSON
  • Publication number: 20180135057
    Abstract: The invention relates to antisense polynucleotide agents targeting the LECT2 gene, and methods of using such antisense polynucleotide agents to inhibit expression of LECT2 and to treat subjects having a LECT2-associated disease, e.g., amyloidosis.
    Type: Application
    Filed: April 8, 2016
    Publication date: May 17, 2018
    Applicant: ALNYLAM PHARMACEUTICALS, INC.
    Inventor: Gregory Hinkle
  • Publication number: 20180135058
    Abstract: Cell death is induced and/or cell growth is suppressed for a cell having a mutation in the BRAF gene. A drug suppressing GST-? is comprised as an active ingredient.
    Type: Application
    Filed: April 15, 2016
    Publication date: May 17, 2018
    Inventors: Yoshiro NIITSU, Hiroki NISHITA
  • Publication number: 20180135059
    Abstract: The use of microorganisms to make omega- and/or omega-l-functionalized products through an iterative carbon chain elongation pathway that we call a reverse beta oxidation pathway. The pathway uses omega-functionalized CoA thioesters as primers and acetyl-CoA as the extender unit in a non-decarboxylative Claisen condensation, and then uses beta oxidation or fatty acid synthesis enzymes to complete the cycle, via reductase, dehydratase and reductase reactions. Various termination enzymes that act on the functionalized beta-keto acyl-CoA intermediates of the pathway and produce omega or omega-1 functionalized products. The action of termination enzymes on such intermediates yield a large variety of products.
    Type: Application
    Filed: October 16, 2017
    Publication date: May 17, 2018
    Inventors: Ramon GONZALEZ, Seokjung CHEONG, James M. CLOMBURG
  • Publication number: 20180135060
    Abstract: The present invention relates to a vector for the expression of recombinant proteins, antigens, pathogen-like particles and immunogenic complexes, said vector (pMRKA vector) being produced by modifying the plasmids containing the gene sequence of the T7 promoter of E. coli, this modification being mainly characterized by the substitution of the ampicillin-resistance gene by the kanamycin-resistance gene, and by the insertion of the par sequence (partition sequence which determines the efficient segregation of the plasmids in daughter cells during cell division). Also provided are expression vectors based on the pMRKA plasmid, which additionally comprise at least one of the gene sequences of the exosome of P. abyssi, which vectors are designated pMRKA-EXO, pMRKA-RING and pSUMAC. The invention also provides the vectors additionally comprising gene sequences with immunomodulatory or immunoregulatory activity, preferably the pMRKA-Z-Z-EXO and pMRKA-Z-Z-RING vectors.
    Type: Application
    Filed: April 20, 2015
    Publication date: May 17, 2018
    Applicant: OURO FINO SAUDE ANIMAL LTDA
    Inventors: Celso Raul Romero Ramos, Miryam Marroquin Quelopana
  • Publication number: 20180135061
    Abstract: A synthetic cDNA which encodes a protein wherein at least one optimal or non-optimal codon in a wild type DNA encoding the protein has been replaced respectively with one or more non-optimal codons or optimal codons encoding the same amino acid.
    Type: Application
    Filed: September 22, 2017
    Publication date: May 17, 2018
    Inventors: Jeffery M. Coller, Kristian E. Baker