Patents Issued in March 8, 2018
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Publication number: 20180066220Abstract: Devices and systems for cell culture that include one or more hollow fibers or channels integrated into a chamber are provided. The hollow fibers or channels and/or the chamber are seeded with one or more cell types. Methods of co-culturing two or more cell types in the devices are also provided.Type: ApplicationFiled: September 6, 2017Publication date: March 8, 2018Applicant: Los Alamos National Security, LLCInventors: Pulak Nath, Jen-Huang Huang
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Publication number: 20180066221Abstract: A buffer tank for storing a culture medium for cell culture, includes: a tank main body including a cylindrical inner surface extending in a vertical direction, an inverted conical storage bottom surface formed below the cylindrical inner surface, and a storage space defined by the cylindrical inner surface and the inverted conical storage bottom surface and configured to store the culture medium an injection portion configured to inject the culture medium from the inverted conical storage bottom surface of the tank main body into the storage space; and a discharge portion configured to discharge the culture medium stored in the storage space, wherein the injection portion is configured to inject the culture medium toward a central axis line of the cylindrical inner surface of the tank main body and obliquely upward with respect to the central axis line.Type: ApplicationFiled: September 1, 2017Publication date: March 8, 2018Inventors: Toshifumi KITAHARA, Kunitada HATABAYASHI, Kenichi KAGAWA, Yusuke YODA
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Publication number: 20180066222Abstract: In various embodiments a Massively parallel Single-cell Electroporation Platform (MSEP) for low voltage, high efficiency delivery of extracellular materials into mammalian cells at an ultrahigh throughput of 10 million cells/min on a 1 cm2 chip is provided. In certain embodiments MSEP is realized by a 3D silicon-based device with, e.g., 5,000 short vertical microfluidic channels in parallel. Single cells flowing through these channels are geometrically confined to regions with intense and localized electric fields where cells are electroporated. High efficiency delivery of calcium dyes, large-sized dextran proteins, and plasmids into mammalian cells to establish a range of sizes and compositions have been successfully accomplished with MSEP.Type: ApplicationFiled: August 9, 2017Publication date: March 8, 2018Inventors: TUHIN SUBHRA SANTRA, MICHAEL A. TEITELL, PEI-YU E. CHIOU
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Publication number: 20180066223Abstract: Embodiments of the invention relate to a cell culture incubator having a gas flow regulation system that exerts control over atmospheric parameters within the incubator. Particular embodiments include an enclosed environmental chamber and a control unit operably linked thereto, the control unit having an oxygen module and a pressure module. Control unit embodiments, by way of these modules, are configured to regulate both oxygen partial pressure and total gas pressure within the enclosed environmental chamber. Embodiments of the control unit are adapted (a) to provide instructions to the oxygen module to regulate an oxygen level to an instructed hypoxic oxygen level and (b) to provide instructions to the pressure module to regulate total gas pressure to an instructed positive pressure level. The regulation of oxygen to the instructed hypoxic level prevails despite an oxygen partial pressure-increasing effect of the positive pressure condition associated with the instructed positive pressure level.Type: ApplicationFiled: October 20, 2017Publication date: March 8, 2018Inventors: James LIM, Luke CASSEREAU
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Publication number: 20180066224Abstract: Methods are disclosed for separating beads and cells from a host fluid. The method includes flowing a mixture containing the host fluid, the beads, and the cells through an acoustophoretic device having an ultrasonic transducer including a piezoelectric material driven by a drive signal to create a multi-dimensional acoustic standing wave. A drive signal is sent to drive the at least one ultrasonic transducer to create the multi-dimensional acoustic standing wave. A recirculating fluid stream having a tangential flow path is located substantially tangential to the standing wave and separated therefrom by an interface region. A portion of the cells pass through the standing wave, and the beads are held back from the standing wave in the recirculating fluid stream at the interface region. Also disclosed is an acoustophoretic device having a coolant inlet adapted to permit the ingress of a cooling fluid into the device for cooling the transducer.Type: ApplicationFiled: August 29, 2017Publication date: March 8, 2018Inventors: Bart Lipkens, Rudolf Gilmanshin, Louis Masi, Benjamin Ross-Johnsrud, Erik Miller, Walter M. Presz, JR., Thomas J. Kennedy, III
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Publication number: 20180066225Abstract: The present invention relates to bacterial ghosts, and more particularly, to a method of preparing bacterial ghosts from gram-positive bacteria by hydrochloric acid treatment. Specifically, according to the present invention, when gram-positive bacteria were cultured after being treated with a minimum inhibitory concentration (MIC) of hydrochloric acid capable of inhibiting colony formation of gram-positive bacteria, bacterial ghosts were effectively formed. Since the formed bacterial ghosts have no intracellular proteins or DNA while preserving cell wall integrity, the risk of side effects such as secondary infection caused by bacterial growth when the bacterial ghosts are administered to humans is low. Therefore, the bacterial ghosts prepared from gram-positive bacteria according to the method of the present invention may be effectively used as a vaccine or a foreign antigen carrier for preventing or treating gram-positive bacterial infection.Type: ApplicationFiled: July 13, 2016Publication date: March 8, 2018Applicant: PAICHAI UNIVERSITY INDUSTRY-ACADEMIC COOPERATION FOUNDATIONInventors: Chang Won CHOI, Seong Mi JI, Hyun Jung PARK, Sung OH, Nagarajan VINOD, Han Byul NO
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Publication number: 20180066226Abstract: Provided is a cell culture support having improved cell adhesion and mobility, which includes: a fibrous web which is made by accumulating fibers containing a hydrophilic polymer and a hydrophobic polymer obtained by electrospinning, in which a plurality of pores into which a culture solution is penetrated are formed.Type: ApplicationFiled: May 10, 2016Publication date: March 8, 2018Inventors: In Yong SEO, Seung Hoon LEE, Song Hee KOO, Ji Hyun LEE
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Publication number: 20180066227Abstract: A method of expanding and maintaining human embryonic stem cells (ESCs) in an undifferentiated state by culturing the ESCs in a suspension culture under culturing conditions devoid of substrate adherence is provided. Also provided are a method of deriving ESC lines in the suspension culture and methods of generating lineage-specific cells from ESCs which were expanded in the suspension culture of the present invention.Type: ApplicationFiled: November 16, 2017Publication date: March 8, 2018Applicant: Technion Research & Development Foundation LimitedInventors: Michal AMIT, Joseph ITSKOVITZ-ELDOR
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Publication number: 20180066228Abstract: The application relates to methods of assessing whether an individual has an exhausted CD8+ T cell or lack of CD4+ T cell costimulation phenotype, and the use of such methods in determining an individual's risk of autoimmune disease progression, progression of a chronic infection, not responding to a treatment for a chronic infection, not mounting an effective immune response to vaccination, infection-associated immunopathology, transplant rejection, or cancer progression.Type: ApplicationFiled: November 15, 2017Publication date: March 8, 2018Inventors: Kenneth G.C. Smith, Paul A. Lyons, Eoin F. McKinney
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Publication number: 20180066229Abstract: This invention provides a method for preparing dendritic cells from monocytes with the use of interferon ? via non-adhesive culture. The method for preparing cytotoxic dendritic cells from monocytes comprises subjecting monocytes which were isolated from the peripheral blood to non-adhesive culture in the presence of GM-CSF and pegylated interferon ?, and further conducting non-adhesive culture with the addition of prostaglandin E2 and OK432.Type: ApplicationFiled: March 16, 2016Publication date: March 8, 2018Applicant: SHINSHU UNIVERSITYInventors: Shigetaka SHIMODAIRA, Terutsugu KOYA
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Publication number: 20180066230Abstract: The present disclosure relates to a culture scaffold for promoting differentiation from stem cells or precursor cells into osteoblasts, in which the culture scaffold includes a structure composed of a ridge and a groove, a kit using the culture scaffold, and a method for differentiating stem cells or precursor cells into osteoblasts. The culture scaffold of the present disclosure has an optimal pattern depending on the type of stem cells or precursor cells, thereby improving the osteoblast differentiation potency. In particular, it has a feature of showing excellent osteoblast differentiation potency even if only a small amount of supplementary factors inducing osteoblast differentiation is added. Furthermore, since the osteoblast differentiation potency is not greatly influenced by the change in cell density, it is possible to induce differentiation into osteoblasts without being influenced by the inflammatory environment formed by the inflammatory factors that increase upon cell differentiation.Type: ApplicationFiled: July 7, 2017Publication date: March 8, 2018Inventors: Hang-Rae Kim, Jin-Hee Kim, Bokyung Kim, Youn Sang Kim, Chang su Kim
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Publication number: 20180066231Abstract: The present invention aims to provide a mesenchymal stem cell culture medium suitable for culturing umbilical cord mesenchymal stem cells in particular, among the mesenchymal stem cells. The present invention is of a mesenchymal stem cell culture medium comprising at least 2 kinds of components selected from the group consisting of a PTEN inhibitor, a p53 inhibitor, a p38 inhibitor, an Wnt signal activator and a ROCK inhibitor as well as basal medium for culturing animal cells.Type: ApplicationFiled: February 26, 2016Publication date: March 8, 2018Applicant: ROHTO PHARMACEUTICAL CO., LTD.Inventors: Yoshifumi IKEYAMA, Toru OKUBO, Hiroyuki NISHIDA, Tomohiro TSUDA, Eiko UNO, Masayo YUMOTO
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Publication number: 20180066232Abstract: The invention relates to a process for preparing a non-expanded tissue derivative, that is not subjected to cell proliferation in vitro, comprising a vascular-stromal fraction enriched in stem and multipotent elements, such as pericytes and/or mesenchymal stem cells, or for preparing non-embryonic stem cells obtained from a tissue sample or from said derivative, wherein said tissue derivatives or said cells are subjected to vibrations derived from a heart sound such to control the degree of differentiation or possible differentiation of said stem and multipotent elements into several other types of cells that is to optimize their potency. The invention relates also to a device for carrying out said process, to stem cells obtainable by the process as well as a drug for the regeneration of an animal tissue.Type: ApplicationFiled: September 13, 2017Publication date: March 8, 2018Inventors: Carlo Tremolada, Carlo Ventura, Milford Graves
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Publication number: 20180066233Abstract: The invention relates to a liver organoid, uses thereof and method for obtaining them.Type: ApplicationFiled: July 31, 2017Publication date: March 8, 2018Applicant: Koninklijke Nederlandse Akademie van WetenschappenInventors: Meritxell Huch Ortega, Johannes Carolus
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Publication number: 20180066234Abstract: Methods to form a surface coating and surface pattern, which are based on adsorption of hydrocarbon chains that can be used with imaging optics to visualize macrophage fusion and multinucleated giant cell formation with living specimens are described.Type: ApplicationFiled: September 8, 2017Publication date: March 8, 2018Inventors: James Faust, Tatiana Ugarova, Robert Ros, Wayne Christenson, Kyle Doudrick
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Publication number: 20180066235Abstract: This application is directed generally to foot-and-mouth disease virus (FMDV) 3C proteases that have been modified by mutating a polynucleotide sequence coding for the FMDV 3C protease. The modified FMDV proteases exhibit proteolytic activity on FMDV P1 precursor protein and exhibit a reduction in one or more toxic or inhibitory properties associated with an unmodified FMDV 3C protease on a host cell used to recombinantly produce it. Vectors carrying polynucleotides encoding modified FMDV 3C protease sequences can induce production of FMDV virus-like particles in a host cell when expressed in the host cell. The modified FMDV 3C proteases can generally be used to produce immunogenic FMDV preparations capable of inducing an immune response against FMDV.Type: ApplicationFiled: September 8, 2016Publication date: March 8, 2018Applicant: The Government of the United States of America, as represented by the Secretary of Homeland SecurityInventors: Michael Puckette, Max V. Rasmussen
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Publication number: 20180066236Abstract: The present invention relates to novel infectious bronchitis virus strains and the uses thereof. The invention particularly relates to an inactivated or attenuated IBV, as well as to vaccine compositions comprising the same and the uses thereof to vaccinate avians. The invention also relates to nucleic acids, infected cells and methods for detecting the infectious bronchitis virus strains of the invention in any sample.Type: ApplicationFiled: October 27, 2017Publication date: March 8, 2018Inventors: WIL SOLANO, BRIANNA FORD, CHRIS LUTHER
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Publication number: 20180066237Abstract: A method of purifying a viral vector is provided. The method includes loading a nonionic density gradient into a continuous flow centrifuge rotor; loading a material including the viral vector and a predictable contaminant into the continuous flow centrifuge rotor; rotating the continuous flow centrifuge rotor in a manner sufficient to separate the viral vector from the predictable contaminant and the material in the nonionic density gradient; and unloading the separated viral vector from the continuous flow centrifuge rotor.Type: ApplicationFiled: November 7, 2017Publication date: March 8, 2018Applicants: ALFA WASSERMANN, INC., Virovek, Inc.Inventors: Sandra Patricia MERIÑO, Haifeng CHEN
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Publication number: 20180066238Abstract: Described herein are mutant pneumoviruses comprising a nucleotide sequence which encodes a mutated zinc binding motif in an M2-1 protein of the pneumovirus, wherein the zinc binding motif is mutated relative to wild-type pneumovirus. The mutant pneumoviruses described herein grow to high titer in cell culture, are genetically stable, are attenuated in vitro and in vivo, and are highly immunogenic. Also described herein are vaccines and vaccine compositions comprising the live attenuated mutant pneumoviruses. Vaccine compositions can further comprise a pharmaceutically acceptable carrier, vehicle, excipient, and/or adjuvant. Methods for inducing a protective immune response in a subject against a pneumovirus infection are also described and disclosed.Type: ApplicationFiled: February 22, 2016Publication date: March 8, 2018Inventors: Mark E. Peeples, Jianrong Li, Hui Cai, Rongzhang Wang
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Publication number: 20180066239Abstract: The disclosure relates to engineered ketoreductase polypeptides and processes of using the polypeptides for production of phenylephrine.Type: ApplicationFiled: November 14, 2017Publication date: March 8, 2018Inventors: Oscar Alvizo, Steven J. Collier, Hans-Georg Joerg Hennemann, Seong Ho Oh, Wenjuan Zha
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Publication number: 20180066240Abstract: The present invention relates to proteins involved in fatty acid synthesis, such as fatty acid synthases (FAS) variants, comprising one or more polypeptide chains, wherein said polypeptide chain(s) comprise one or more subunits comprising a malonyl/palmitoyl transferase domain (MPT domain), acetyl transferase domain (AT domain), and ketoacyl synthase domain (KS domain), and at least one amino acid substitution in the MPT domain at a position corresponding to R130, in the AT domain at a position corresponding to I306, and/or in the KS domain, preferably in the acyl binding channel and/or at KS domain binding site to ACP, to modulate affinities of acyl intermediates, and optionally further amino acid substitution(s). The present invention relates to the respective polypeptide domains. The present invention further relates to nucleic acid molecules encoding the proteins (or the polypeptide domains) and to host cells containing said nucleic acid molecules.Type: ApplicationFiled: March 31, 2016Publication date: March 8, 2018Inventors: Martin Grininger, Jan Gajewski, Eckhard Boles, Renata Pavlovic
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Publication number: 20180066241Abstract: The invention provide chimeric peptides comprising a chimeric peptide comprising a cell-penetrating peptide linked to a pro-apoptotic peptide, wherein the pro-apoptotic peptide is derived from, or consists of, a portion of PP2A protein that binds a SET protein or is derived from, or consists of, a portion of the SET protein that binds PP2A protein.Type: ApplicationFiled: March 31, 2016Publication date: March 8, 2018Inventors: Angelita Rebollo Garcia, Jeronimo Bravo Sicilia, Jesus Maria Fominaya Gutierrez, Etienne Waelkens, Veerle Janssens
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Publication number: 20180066242Abstract: The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends the engineering of optimized modular CRISPR-Cas enzyme systems.Type: ApplicationFiled: June 10, 2016Publication date: March 8, 2018Inventors: Feng Zhang, Bernd ZETSCHE
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Publication number: 20180066243Abstract: The invention relates to a host cell comprising at least four different heterologous polynucleotides chose from the group of polynucleotides encoding cellulases, hemicellulases and pectinases, wherein the host cell is capable of producing the at least four different enzymes chosen from the group of cellulases, hemicellulases and pectinases, wherein the host cell is a filam-tous fungus and is capable of secretion of the at least four different enzymes. This host cell can suitably be used for the production of an enzyme composition that can be use in a process for the saccharification of cellulosic material.Type: ApplicationFiled: November 16, 2017Publication date: March 8, 2018Inventors: Alrik Pieter LOS, Cornelis Maria Jacobus Sagt, Margot Elisabeth Francoise Schooneveld-Bergmans, Robbertus Antonius Damveld
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Publication number: 20180066244Abstract: The present invention provides methods and compositions comprising at least one neutral metalloprotease enzyme that has improved storage stability. In some embodiments, the neutral metalloprotease finds use in cleaning and other applications. In some particularly preferred embodiments, the present invention provides methods and compositions comprising neutral metalloprotease(s) obtained from Bacillus sp. In some more particularly preferred embodiments, the neutral metalloprotease is obtained from B. amyloliquefaciens. In still further preferred embodiments, the neutral metalloprotease is a variant of the B. amyloliquefaciens neutral metalloprotease. In yet additional embodiments, the neutral metalloprotease is a homolog of the the B. amyloliquefaciens neutral metalloprotease. The present invention finds particular use in applications including, but not limited to cleaning, bleaching and disinfecting.Type: ApplicationFiled: October 19, 2017Publication date: March 8, 2018Inventors: ANDREW SHAW, DAVID A ESTELL, EUGENE S SADLOWSKI, HIROSHI OH, LOUISE WALLACE, RONALDUS WILHELMUS JOANNES HOMMES, SANG-KYU LEE
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Publication number: 20180066245Abstract: One aspect of the present invention relates to mutants of chondroitinase ABCI. Such chondroitinase ABCI mutants exhibit altered chondroitin lyase activity or increased resistance to inactivation from stressors including exposure to UV light or heat. Methods of using chondroitinase ABCI mutant enzymes are also provided.Type: ApplicationFiled: October 20, 2017Publication date: March 8, 2018Inventors: Anthony O. CAGGIANO, Jennifer IACI, Andrea VECCHIONE, Elizabeth HUNTER
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Publication number: 20180066246Abstract: Methods of isolating nucleic acids comprising DNA from biological material are disclosed. The methods comprise a lysis step using an aqueous composition which comprises lithium at a concentration of 0.05 to 1.0M, a chelating agent, and a surfactant to produce a lysed composition. The lysed composition is treated with a solid support that is capable of immobilising DNA in the presence of a dissolved chaotropic agent at a concentration of 0.05 to 2M and 25% to 60% by volume of a C1-3 alkanol. The support is then washed with a first wash solution containing lithium dissolved in a C1-3 alkanol. Subsequently, the support is washed with a liquid comprising at least 80% by volume of a C1-3 alkanol. The nucleic acid comprising DNA is then eluted from the support.Type: ApplicationFiled: March 17, 2016Publication date: March 8, 2018Inventors: Stephen John MINTER, Georgios PATSOS
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Publication number: 20180066247Abstract: According to one embodiment, a biological tissue grinding container includes a container portion and a vibrated portion, and vibration is transmitted to biological tissue from the vibration portion to grind the biological tissue. The vibrated portion includes a contact portion to be brought into direct contact with the biological tissue and defines a chamber which stores the biological tissue to be ground, together with the container portion. The vibrated portion is fixedly supported to be vibratable to the container portion directly or via a support portion provided between the container portion and itself, and has solidity higher than that of the container portion or support portion.Type: ApplicationFiled: September 6, 2017Publication date: March 8, 2018Applicant: Toshiba Medical Systems CorporationInventors: Hiroaki Kushima, Nobuo Kawamura, Tetsuya Kuwabara
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Publication number: 20180066248Abstract: A system, configured to facilitate processing and detection of nucleic acids, the system comprising a process fluid container and a cartridge comprising: a top layer, a set of sample port-reagent port pairs, a shared fluid port, a vent region, a heating region, and a set of detection chambers; an intermediate substrate, coupled to the top layer comprising a waste chamber; an elastomeric layer, partially situated on the intermediate substrate; and a set of fluidic pathways, each formed by at least a portion of the top layer and a portion of the elastomeric layer, wherein each fluidic pathway is fluidically coupled to a sample port-reagent port pair, the shared fluid port, and a detection chamber, comprises a portion passing through the heating region, and is configured to be occluded upon deformation of the elastomeric layer, to transfer a waste fluid to the waste chamber, and to pass through the vent region.Type: ApplicationFiled: November 9, 2017Publication date: March 8, 2018Inventors: Michael T. Kusner, Sundaresh Brahmasandra, Jeffrey Williams, Robert Jaekel
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Publication number: 20180066249Abstract: The invention relates to methods and kits for labeling and manipulating a cellular circuit. The methods includes transfecting a first-order cell in the cellular circuit with a nucleic acid molecule encoding a tethered ligand, and a second-order cell in the cellular circuit with a nucleic acid molecule encoding a receptor and an effector fusion polypeptide, a nucleic acid molecule encoding a receptor interactor and protease fusion polypeptide, and a nucleic acid molecule encoding a reporter/modifier gene.Type: ApplicationFiled: March 15, 2016Publication date: March 8, 2018Inventors: Gilad BARNEA, Mustafa TALAY, Ethan RICHMAN, John SZYMANSKI, Mark JOHNSON, John FISHER, Nathaniel SNELL
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Publication number: 20180066250Abstract: A pharmaceutical composition for preventing and treating endocrine disrupting chemicals-induced diseases and a treating method using the same. Since the composition has an effect of decreasing lipid accumulation caused by endocrine disrupting chemicals, for example, persistent organic pollutants (POPs) including polychlorinated biphenyl and the like and can improve insulin resistance caused by the POPs through reduction of insulin receptor substrate 1 (IR1), the composition can be helpfully used for treating diseases including obesity, insulin resistance, and the like caused by the endocrine disrupting chemicals. Further, according to the present disclosure, the composition has an effect of excreting the endocrine disrupting chemicals.Type: ApplicationFiled: October 27, 2016Publication date: March 8, 2018Inventors: Young Hyun YOO, Hye Young KIM
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Publication number: 20180066251Abstract: The present invention relates to small RNAs, inhibitors thereof, inhibitors of enzymes producing thereof, and their use to modulate the response of a cell to a DNA damaging event. The invention concerns also a method to detect the presence or quantify DNA damage.Type: ApplicationFiled: March 31, 2017Publication date: March 8, 2018Inventors: Fabrizio d'Adda di Fagagna, Sofia Francia, Flavia Michelini, Francesca Rossiello
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Publication number: 20180066252Abstract: Embodiments of the disclosure include methods and compositions for in situ cardiac cell regeneration, including transdifferentiation of cardiac cells to cardiomyocytes. In particular embodiments, in situ cardiac cell regeneration encompasses delivery of p63 shRNA and one or both of Hand2 and myocardin, and in specific embodiments further includes one or more of Gata4, Mef2c, and Tbx5. In specific aspects of the disclosure, adult cardiac fibroblasts are reprogrammed into cardiomyocytes using viral vectors that harbor p63 shRNA and one or both of the transcription factors Hand2 and myocardin.Type: ApplicationFiled: February 19, 2016Publication date: March 8, 2018Inventors: Vivekkumar B. Patel, Hongran Wang, Vivek P. Singh, Erin Lynn Reineke, Megumi Mathison, Austin J. Cooney, Todd Rosengart
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Publication number: 20180066253Abstract: The present invention includes methods and compositions for modifying endothelial cells for improved vascular self-assembly. In some embodiments, the invention includes a modified endothelial cell and methods of generating and using the modified endothelial cell in a pharmaceutical composition. Other embodiments include methods of promoting vascular self-assembly and regenerating vascular structures in a subject in need thereof. In particular, the modified endothelial cell has reduced immunogenicity in an allogeneic environment in the subject, while still capable of forming vascular structures.Type: ApplicationFiled: March 22, 2016Publication date: March 8, 2018Inventors: Jordan POBER, Parwiz ABRAHIMI
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Publication number: 20180066254Abstract: Therapeutic agent for use in the treatment of neurodegenerative diseases associated with abnormalities of MAPT gene encoded protein tau, wherein said therapeutic agent comprises one or more siRNAs targeting MAPT exon 10 sequence.Type: ApplicationFiled: March 24, 2016Publication date: March 8, 2018Inventors: Giuseppina COVELLO, Michela Alessandra DENTI, Kavitha SIVA
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Publication number: 20180066255Abstract: The invention relates to double-stranded ribonucleic acid (dsRNA) targeting a PROC gene, and methods of using the dsRNA to inhibit expression of PROC.Type: ApplicationFiled: July 6, 2017Publication date: March 8, 2018Inventors: Ivanka Toudjarska, John M. Maraganore, Brian Bettencourt, Stuart Milstein, Martin A. Maier, Klaus Charisse, Kallanthottathil Rajeev, Satyanarayana Kuchimanchi
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Publication number: 20180066256Abstract: The current invention provides an improved oligonucleotide and its use for treating, ameliorating, preventing, delaying and/or treating a human cis-element repeat instability associated genetic neuromuscular or neurodegenerative disorder.Type: ApplicationFiled: August 14, 2017Publication date: March 8, 2018Inventors: Peter Christian de Visser, Susan Allegonda Maria Mulders
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Publication number: 20180066257Abstract: An siRNA for specifically targeting a PMP22 mutant gene and a pharmaceutical composition for preventing or treating Charcot Marie Tooth disease, which includes the same, are provided. According to the present invention, it is confirmed that selective suppression of a PMP22 mutant allele by a non-viral delivery system of siRNA may improve demyelinating neuropathic symptoms of CMT in vivo, enhance a motor ability and increase a volume of muscle. Therefore, the siRNA may be used in a useful method for treating various dominantly inherited peripheral neuropathies including CMT.Type: ApplicationFiled: August 30, 2017Publication date: March 8, 2018Inventors: Byung-Ok CHOI, Young Bin HONG
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Publication number: 20180066258Abstract: The invention provides specific synthetic chimeric xenonucleic acid guide RNA; s(XNA-gRNA) for enhancing crispr mediated genome editing efficiency. The invention also provides methods and compositions for inducing CRISPR/Cas-based gene editing/regulation (e.g., genome editing or gene expression) of a target nucleic acid (e.g., target DNA or target RNA) in a cell. The methods include using single guide RNAs (sgRNAs) that have been chemically modified with xeno nucleic acids which enhance gene regulation of the target nucleic acid in a primary cell for use in ex vivo therapy or in a cell in a subject for use in in vivo therapy. Additionally, provided herein are methods for preventing or treating a genetic disease in a subject by administering a sufficient amount of a sgRNA that has been chemically modified with xeno nucleic acids to correct a mutation in a target gene associated with the genetic disease.Type: ApplicationFiled: October 17, 2017Publication date: March 8, 2018Inventor: Michael J Powell
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Publication number: 20180066259Abstract: Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.Type: ApplicationFiled: October 20, 2017Publication date: March 8, 2018Inventors: Peter SAZANI, Ryszard KOLE
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Publication number: 20180066260Abstract: The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Interferon Regulatory Factor 8 (IRF8), in particular, by targeting natural antisense polynucleotides of Interferon Regulatory Factor 8 (IRF8). The invention also relates to the identification of these antisense oligonucleotides and their use in treating diseases and disorders associated with the expression of IRF8.Type: ApplicationFiled: October 31, 2017Publication date: March 8, 2018Inventors: Joseph COLLARD, Olga KHORKOVA SHERMAN
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Publication number: 20180066261Abstract: The present disclosure relates generally to cancer and particularly to breast cancer including estrogen sensitive, estrogen resistant and triple negative breast cancer (TNBC), and to methods of diagnosis and prognosis thereof and therapeutic intervention involving replication factor C 40 (RFC40). Methods and assays for evaluating breast cancer are provided. The disclosure also relates to inhibition or modulation of RFC40 in treatment or alleviation of cancer, including breast cancer. RFC40 inhibitors, including siRNAs, miRNAs, and shRNAs, which specifically affect cancer cells, particularly breast cancer cells, are provided.Type: ApplicationFiled: November 20, 2017Publication date: March 8, 2018Inventor: Rakhee S. Gupte
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Publication number: 20180066262Abstract: Methods and compositions are provided for oligonucleotide probes and oligonucleotide probe libraries that recognize targets of interest. The targets include circulating biomarkers such as microvesicles, including those derived from various diseases.Type: ApplicationFiled: March 9, 2016Publication date: March 8, 2018Inventors: Valeriy DOMENYUK, Adam STARK, Nianqing XIAO, Zhenyu ZHONG, Mark MIGLARESE, David SPETZLER
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Publication number: 20180066263Abstract: [Problem] To provide a novel nucleic acid aptamer for a vascular endothelial growth factor receptor, said nucleic acid aptamer being useful for the diagnosis and treatment of various diseases associated with VEGFs that can regulate angiogenesis and receptors for the VEGFs, e.g., tumor angiogenesis, diabetic retina and chronic rheumatoid arthritis. [Solution] A nucleic acid aptamer characterized by comprising a nucleotide sequence represented by any one of SEQ ID NOs: 1 to 5, and also characterized by being capable of bonding to a human VEGF receptor specifically. In a preferred embodiment of the nucleic acid aptamer, a primer recognition sequence, a fluorescent label, or a biotin molecule, an avidin molecule, a streptavidin molecule or other specific binding tag peptide may be linked to the 5?- or 3?-terminal of the nucleic acid aptamer for the purpose of making it possible to detect the nucleic acid aptamer easily.Type: ApplicationFiled: March 28, 2016Publication date: March 8, 2018Applicants: NISSAN CHEMICAL INDUSTRIES, LTD., THE UNIVERSITY OF TOKYOInventors: Keitaro YOSHIMOTO, Keiko KIMURA, Hitoshi FURUSHO
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Publication number: 20180066264Abstract: 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, such as THC.Type: ApplicationFiled: November 13, 2017Publication date: March 8, 2018Inventor: George W. Jackson
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Publication number: 20180066265Abstract: The present invention is related to a nucleic acid, preferably binding to MCP-1, selected from the group comprising type 1A nucleic acids, type 1B nucleic acids, type 2 nucleic acids, type 3 nucleic acids, type 4 nucleic acids and nucleic acids having a nucleic acid sequence according to any of SEQ.ID.No. 87 to 115.Type: ApplicationFiled: June 6, 2017Publication date: March 8, 2018Inventors: Werner Purschke, Florian Jarosch, Dirk Eulberg, Sven Klussmann, Klaus Buchner, Christian Maasch
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Publication number: 20180066266Abstract: The present inventors found that a fusion gene present in some cancer patients is an oncogene. The present invention relates to a polypeptide as a novel fusion protein, a polynucleotide encoding the polypeptide, a vector comprising the polynucleotide, a transformed cell comprising the vector, a method for detecting the fusion protein or polynucleotide, a method for screening a therapeutic agent for cancer, and a method for treating cancer that is shown to be positive for the fusion gene.Type: ApplicationFiled: November 13, 2017Publication date: March 8, 2018Applicants: Astellas Pharma Inc., CureGene K.K.Inventors: HIROYUKI MANO, Sadao Kuromitsu, Nobuaki Shindo, Takatoshi Soga, Takashi Furutani
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Publication number: 20180066267Abstract: Polynucleotides encoding fusion proteins comprising interferons and luciferases which have biotherapeutic, diagnostic, and quality control applications in biotechnological, medical, and veterinary fields.Type: ApplicationFiled: May 1, 2017Publication date: March 8, 2018Applicant: The Government of the United States of America, as represented by the Secretary of Homeland SecurityInventors: Michael Puckette, Max V. Rasmussen
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Publication number: 20180066268Abstract: Disclosed are eukaryotic expression systems and methods for the selection of mammalian cell lines that produce proteins of interest, such as therapeutic proteins. The systems and methods allow for a simple and fast selection of cells mediating high levels of recombinant protein production. The systems and methods decrease the efforts and time needed to bring a new therapeutic protein to the patients, and also lower the cost of the therapeutic protein by increasing the productivity of cells in a bioreactor.Type: ApplicationFiled: April 1, 2016Publication date: March 8, 2018Inventors: Nicolas Mermod, Lucille Pourcel, Pierre-Alain Girod, Valerie Le Fourn
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Publication number: 20180066269Abstract: Provided are a novel promoter and a method of producing a target product using the same.Type: ApplicationFiled: January 15, 2016Publication date: March 8, 2018Inventors: Seung Bin LEE, Hyun Ae BAE, Ji Hye LEE, Young Lyeol YANG