The Polynucleotide Is Encapsidated Within A Virus Or Viral Coat Patents (Class 435/456)
  • Patent number: 10294278
    Abstract: Provided herein is a nucleic acid comprising consensus amino acid sequence of foot-and-mouth disease FMDV VP1-4 coat proteins of FMDV subtypes A, Asia 1, C, O, SAT1, SAT2, and SAT3 as well as plasmids and vaccines expressing the sequences. Also provided herein is methods for generating an immune response against one or more FMDV subtypes using the vaccine as described above as well as methods for deciphering between vaccinated mammals with the vaccine and those that are infected with FMDV.
    Type: Grant
    Filed: August 3, 2015
    Date of Patent: May 21, 2019
    Inventors: David B. Weiner, Bernadette Ferraro, Jian Yan, Patricia A. Brown, Rodney A. Bowling, Douglas R. Kern, Mathura P. Ramanathan, Niranjan Y. Sardesai, Karuppiah Muthumani
  • Patent number: 10265417
    Abstract: Sequences of novel adeno-associated virus capsids and vectors and host cells containing these sequences are provided. Also described are methods of using such host cells and vectors in production of rAAV particles. AAV-mediated delivery of therapeutic and immunogenic genes using the vectors of the invention is also provided.
    Type: Grant
    Filed: August 3, 2016
    Date of Patent: April 23, 2019
    Assignee: The Trustees of the University of Pennsylvania
    Inventors: James M. Wilson, Guangping Gao, Mauricio R. Alvira, Luc H. Vandenberghe
  • Patent number: 10226486
    Abstract: The present invention relates to a method for preparing induced dopaminergic neuronal progenitors (iDPs) comprising inducing an expression of Oct4, Sox2, Klf4, and c-Myc genes in adult cells and direct reprogramming of the adult cells to the iDPs by treating the cells with sonic hedgehog (SHH) and fibroblast growth factor 8 (FGF8); and a cell therapy product and a composition for treating or preventing Parkinson's Disease (PD) which comprises the iDPs as active ingredients. In addition to this, the present invention relates to a method for preparing midbrain dopaminergic neurons comprising isolating NSC-like colony by culturing the iDPs, dissociating the isolated NSC-like colony, and culturing the cells on the neural cell differentiation medium. Further, the present invention relates to a method for treating PD or a method for screening medicine for preventing or treating PD using the cells of the present invention.
    Type: Grant
    Filed: October 4, 2013
    Date of Patent: March 12, 2019
    Assignee: KOREA RESEARCH INSTITUTE OF BIOSCIENCE AND BIOTECHNOLOGY
    Inventors: Yee Sook Cho, Jang Hwan Kim, Han Seop Kim
  • Patent number: 10214566
    Abstract: The present invention provides mutant adeno-associated virus (AAV) that exhibit altered capsid properties, e.g., reduced binding to neutralizing antibodies in serum and/or altered heparin binding and/or altered infectivity of particular cell types. The present invention further provides libraries of mutant AAV comprising one or more mutations in a capsid gene. The present invention further provides methods of generating the mutant AAV and mutant AAV libraries, and compositions comprising the mutant AAV. The present invention further provides recombinant AAV (rAAV) virions that comprise a mutant capsid protein. The present invention further provides nucleic acids comprising nucleotide sequences that encode mutant capsid proteins, and host cells comprising the nucleic acids. The present invention further provide methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.
    Type: Grant
    Filed: August 5, 2016
    Date of Patent: February 26, 2019
    Assignee: The Regents of the University of California
    Inventors: David V. Schaffer, Brian Kaspar, Narendra Maheshri
  • Patent number: 10208318
    Abstract: The present invention relates to compositions and methods for the delivery of therapeutic proteins to the CNS using recombinant AAV vectors. More specifically, the invention relates to compositions and methods for delivering proteins into the cerebrospinal fluid of mammalian subjects through peripheral administration of AAV vectors. The invention may be used to treat various disorders of the central nervous system, including degenerative diseases and motor neuron diseases.
    Type: Grant
    Filed: July 22, 2008
    Date of Patent: February 19, 2019
    Assignees: GENETHON, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS)
    Inventor: Martine Barkats
  • Patent number: 10072245
    Abstract: The present invention provides a method for generation of a cell composition of mesencephalic dopaminergic progenitor cells from a starting cell composition comprising pluripotent and/or multipotent stem cells, the method comprising the steps of a) differentiating said pluripotent and/or multipotent stem cells into mesencephalic dopaminergic progenitor cells, thereby generating a cell population comprising mesencephalic dopaminergic progenitor cells and other cells, b) dissociating the differentiated cells of step a) into a single cell suspension, and c) enriching said mesencephalic dopaminergic progenitor cells by using an antigen binding molecule specific for the CD47 antigen for positive selection of said mesencephalic dopaminergic progenitor cells in said single cell suspension. Said method may be performed in a closed cell sample processing system and may be performed in an automated manner.
    Type: Grant
    Filed: February 26, 2016
    Date of Patent: September 11, 2018
    Assignee: Miltenyi Biotec GmbH
    Inventors: Andreas Bosio, Sebastian Knobel, Daniela Lehnen, Serena Barral
  • Patent number: 10036009
    Abstract: The invention relates to methods for isolating traffic-enhancing mutants of drug delivery proteins. In one embodiment, the invention provides a carrier for delivering a therapeutic agent to an organelle, comprising a polypeptide encoded by a mutant penton base gene. In another embodiment, the invention provides a method of enhancing trafficking to a cell by administering a composition comprising a penton base (PB) protein with one or more mutations that enhance cellular entry.
    Type: Grant
    Filed: August 2, 2013
    Date of Patent: July 31, 2018
    Assignee: CEDARS-SINAI MEDICAL CENTER
    Inventor: Lali K. Medina-Kauwe
  • Patent number: 9856295
    Abstract: The present invention provides vectors that contain and express in vivo or in vitro FeLV antigens that elicit an immune response in animal or human against FeLV, compositions comprising said vectors and/or FeLV polypeptides, methods of vaccination against FeLV, and kits for use with such methods and compositions.
    Type: Grant
    Filed: October 6, 2014
    Date of Patent: January 2, 2018
    Assignee: MERIAL INC.
    Inventors: Herve Poulet, Thierry Heidmann
  • Patent number: 9833480
    Abstract: The invention features nucleic acid constructs encoding chimeric immune T-cell receptors (CIRs) that are useful for treating HIV in patients. In general, the CIRs contain an extracellular domain which targets HIV or HIV infected cells (e.g., the extracellular domain of CD4), a transmembrane domain, and a cytoplasmic domain for mediating T-cell activation (e.g., CD3 zeta and/or the partial extracellular domain of CD28). The invention also features the use of host cells expressing CIRs in the treatment of HIV.
    Type: Grant
    Filed: April 14, 2011
    Date of Patent: December 5, 2017
    Assignee: Prospect Chartercare, LLC
    Inventors: Richard P. Junghans, Nithianandan Selliah
  • Patent number: 9822338
    Abstract: Methods, compositions and kits for producing functional neurons, astroctyes, oligodendrocytes and progenitor cells thereof are provided. These methods, compositions and kits find use in producing neurons, astrocytes, oligodendrocytes, and progenitor cells thereof for transplantation, for experimental evaluation, as a source of lineage- and cell-specific products, and the like, for example for use in treating human disorders of the CNS. Also provided are methods, compositions and kits for screening candidate agents for activity in converting cells into neuronal cells, astrocytes, oligodendrocytes, and progenitor cells thereof.
    Type: Grant
    Filed: May 13, 2015
    Date of Patent: November 21, 2017
    Assignee: The Board of Trustees of the Leland Stanford Junior University
    Inventors: Marius Wernig, Thomas C. Sudhof, Thomas Vierbuchen, Austin Ostermeier, Zhiping Pang
  • Patent number: 9816108
    Abstract: Disclosed are viral vector compositions comprising polynucleotide sequences that express one or more biologically-active mammalian guanylate cyclase proteins. Also disclosed are methods for their use in preventing, treating, and/or ameliorating at least one or more symptoms of a disease, disorder, abnormal condition, or dysfunction resulting at least in part from a guanylate cyclase deficiency in vivo. In particular embodiments, the use of recombinant adeno-associated viral (rAAV) vectors to treat or ameliorate symptoms of Leber's congenital amaurosis, as well as other conditions caused by an absence or reduction in the expression of a functional retinal-specific guanylate cyclase 1 (retGC1).
    Type: Grant
    Filed: April 22, 2011
    Date of Patent: November 14, 2017
    Assignee: University of Florida Research Foundation, Inc.
    Inventors: Shannon Elizabeth Boye, William W. Hauswirth, Sanford Leon Boye
  • Patent number: 9783824
    Abstract: The teachings herein are generally directed to a method of enhancing the genetic stability of parvovirus vectors. The stability of conventional ss or dsAAV vector constructs can be enhanced, for example, to obtain a concurrent increase in vector titer and purity, as well as an improvement in vector safety, due at least in part to the elimination of stuffer DNA from the AAV vector. The method is broadly applicable to all gene transfer/therapy applications, such as those requiring delivery of foreign DNA containing recombinant gene expression cassettes. Such foreign DNA can range, for example, from about 0.2 up to about 5.2 kb in length. The enhanced vector constructs are highly flexible, user-friendly, and can be easily modified (via routine DNA cloning) and utilized (via standard AAV vector technology) by anyone skilled in the art.
    Type: Grant
    Filed: September 1, 2015
    Date of Patent: October 10, 2017
    Assignee: The Board of Trustees of the Leland Stanford Junior University
    Inventors: Mark A. Kay, Dirk Grimm
  • Patent number: 9775898
    Abstract: Vaccine compositions for use in inducing enhanced antigen-specific T cell-mediated immune responses in a subject in need thereof are disclosed. The composition comprises (a) a therapeutically effective amount of an immunogenic protein comprising at least an antigen of a pathogen; (b) a saponin-base adjuvant selected from the group consisting of GPI-0100, Quil A, QS-21; and (c) a Toll-like receptor (TLR) agonist adjuvant selected from the group consisting of monophosphoryl lipid A (MPL), and CpG1826.
    Type: Grant
    Filed: February 24, 2016
    Date of Patent: October 3, 2017
    Assignee: TheVax Genetics Vaccine Co., Ltd.
    Inventors: Chia-Mao Wu, Jiun-Ming Wu, Yi-Tsui Chiu, Yin-Ching Lin, Hsien-Kai Chuang, Fu-Tan Hsieh, Kuan-Ming Chen
  • Patent number: 9717759
    Abstract: Disclosed herein are methods and compositions for modulating activity of CXCR4 genes, for example using zinc finger transcription factors (ZF-TFs) or zinc finger nucleases (ZFNs) comprising a zinc finger protein and a cleavage domain or cleavage half-domain. Polynucleotides encoding ZF-TFs or ZFNs, vectors comprising polynucleotides encoding ZF-TFs or ZFNs and cells comprising polynucleotides encoding ZF-TFs or ZFNs and/or cells comprising ZF-TF or ZFNs are also provided.
    Type: Grant
    Filed: September 24, 2014
    Date of Patent: August 1, 2017
    Assignee: Sangamo Therapeutics, Inc.
    Inventors: Michael C. Holmes, Jeffrey C. Miller, Jianbin Wang
  • Patent number: 9719107
    Abstract: The embodiments disclosed herein relate to the construction of fully-deleted Adenovirus-based gene delivery vectors packaged without helper Adenovirus, and more particularly to their use in gene therapy for gene and protein expression, vaccine development, and immunosuppressive therapy for allogeneic transplantation. In an embodiment, a method for propagating an adenoviral vector includes (a) providing an Adenovirus packaging cell line; (b) transfecting a fully-deleted Adenoviral vector construct into the cell line; and optionally (c) transfecting a packaging construct into the cell line, wherein the fully-deleted Adenoviral vector construct and optionally the packaging construct can transfect the Adenovirus packaging cell line resulting in the encapsidation of a fully-deleted Adenoviral vector independent of helper Adenovirus. In an embodiment, a target cell is transduced with the encapsidated fully-deleted Adenoviral vector for treating a condition, disease or a disorder.
    Type: Grant
    Filed: October 23, 2015
    Date of Patent: August 1, 2017
    Assignee: Isogenis, Inc.
    Inventors: Miles B. Brennan, Erin K. Spiegel, Uwe D. Staerz, Charles Wall, Janae Wheeler, William J. Maslanik, Xianghua Zhang
  • Patent number: 9714434
    Abstract: The present invention relates to a vector particle for transferring biological material into cells, wherein said vector particle comprises at least: a first protein which comprises the transmembrane and extracellular domains of the feline endogenous RD114 virus envelope glycoprotein, and a second protein which comprises a ligand of the c-Kit receptor.
    Type: Grant
    Filed: June 10, 2013
    Date of Patent: July 25, 2017
    Assignee: INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
    Inventors: Francois-Loic Cosset, Els Verhoeyen, Caroline Costa, Cecilia Frecha
  • Patent number: 9683252
    Abstract: A method for producing a protein of interest on a manufacturing scale is based on integration, by homologous recombination, of the DNA encoding the protein of interest into a bacterial cell genome at a pre-selected site. The manufacturing scale production of recombinant proteins is in the fed-batch mode, semi-continuous or in a chemostat.
    Type: Grant
    Filed: May 16, 2008
    Date of Patent: June 20, 2017
    Assignees: BOEHRINGER INGELHEIM RCV GMBH & CO KG, SANDOZ AG
    Inventors: Gerald Striedner, Johann Huber, Daniela Keller
  • Patent number: 9637732
    Abstract: Provided is a method of improving the efficiency of iPS cell establishment, comprising bringing one or more factors selected from the group consisting of proteins belonging to cyclin D family and nucleic acids that encode the same into contact with a somatic cell, in the step of nuclear reprogramming of the somatic cell. Also provided are a method of producing an iPS cell comprising the step of bringing the factor(s) and nuclear reprogramming substance(s) into contact with a somatic cell, an iPS cell comprising a nucleic acid that encodes a protein belonging to cyclin D family that can be obtained by the method of producing an iPS cell, and a method of somatic cell production by forcing the iPS cell to differentiate.
    Type: Grant
    Filed: November 4, 2011
    Date of Patent: May 2, 2017
    Assignee: Kyoto University
    Inventors: Shinya Yamanaka, Kazutoshi Takahashi, Koji Tanabe, Hong Hyenjong
  • Patent number: 9506082
    Abstract: The present invention relates to recombinant DNA molecules such as plasmids, non-viral vectors, viral vectors and hybrids thereof, and more particularly to vector modifications that improve expression of said DNA molecules in cell lines and organisms.
    Type: Grant
    Filed: April 8, 2011
    Date of Patent: November 29, 2016
    Assignee: Nature Technology Corporation
    Inventor: James A. Williams
  • Patent number: 9498519
    Abstract: The present invention relates to a pharmaceutical composition for preventing or treating an amyloid ?-caused disease, which comprises as an active ingredient a NIa (nuclear inclusion a) protease or a gene carrier containing a nucleotide sequence encoding the NIa protease. The pharmaceutical composition of this invention is very effective to treat a variety of diseases or disorders, inter alia, Alzheimer's disease.
    Type: Grant
    Filed: July 23, 2010
    Date of Patent: November 22, 2016
    Assignee: GWANGJU INSTITUTE OF SCIENCE AND TECHNOLOGY
    Inventors: Woo Jin Park, Hye-eun Han
  • Patent number: 9492510
    Abstract: Polynucleotides encoding a secreted mutant human carboxylesterase enzyme and polypeptides encoded by the polynucleotides which are capable of metabolizing a prodrug and inactive metabolites thereof to active drug are provided. Compositions and methods for sensitizing cells to a prodrug agent, inhibiting cell growth, treating drug addiction, and facilitating the metabolism of an organophosphate with this enzyme are also provided. In addition, a screening assay for identification of drugs activated by this enzyme is described.
    Type: Grant
    Filed: February 3, 2015
    Date of Patent: November 15, 2016
    Assignees: St. Jude Children's Research Hospital, The University of North Carolina
    Inventors: Philip M. Potter, Monika Weirdl, Matthew R. Redinbo
  • Patent number: 9493743
    Abstract: The present invention relates to a method for preparing commercial scale quantities of human functional beta cells and to the establishment of cell lines from non-foetal pancreatic tissues. It also relates to a method of diagnosis using beta cell tumors or cells derived thereof. The method comprises sub-transplantation procedure to enrich the graft in proliferating beta cells, allowing to generate human Beta cell lines. Such lines express, produce and secrete insulin upon glucose stimulation. They have a gene expression profile that resembles to adult beta cells. In addition, the human beta cell lines are able to normalize glycemia of diabetic mice when transplanted, demonstrating their insulin secretion capabilities.
    Type: Grant
    Filed: October 18, 2012
    Date of Patent: November 15, 2016
    Assignees: UNIVERCELL BIOSOLUTIONS, CENTRE NATIONAL DE LA RECHERCHE SCIENTIFIQUE (CNRS), INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
    Inventors: Paul Czernichow, Philippe Ravassard, Raphael Scharfmann
  • Patent number: 9492568
    Abstract: Disclosed is a method for suppressing the growth of a target cell, which is not limited in the type of a target cell and the type of a protein to be expressed in the target cell and needs not any preparatory experiment for determining a codon to be contained in a protein to be expressed in a target cell. Specifically disclosed is a method for suppressing the growth of a target cell, which comprises the steps of: incorporating DNA containing a region encoding a protein into the target cell, and allowing a protein encoded by the DNA to be expressed in the target cell into which the DNA has been incorporated. The region contained in the DNA comprises a tri-nucleotide sequence. The tri-nucleotide sequence is selected from codons that define at least some amino acid species constituting the protein, and is complementary to at least some codons that are used in the target cell at a frequency of 0.2 or less.
    Type: Grant
    Filed: December 2, 2009
    Date of Patent: November 15, 2016
    Assignee: JAPAN AGENCY FOR MARINE-EARTH SCIENCE AND TECHNOLOGY
    Inventor: Hideki Kobayashi
  • Patent number: 9481714
    Abstract: A fusion protein for use as an immunogen enhancer for enhancing antigen-specific T cell responses is disclosed. The fusion protein comprises: (a) an antigen-presenting cell (APC)-binding domain or a CD91 receptor-binding domain; (b) a protein transduction domain; and (c) an antigen of a pathogen, wherein the APC-binding domain or the CD91 receptor-binding domain is located at the N-terminus of the fusion protein, and the antigen of the pathogen is located at the C-terminus of the protein transduction domain. The protein transduction domain is selected from the group consisting of: (i) a fusion polypeptide, comprising a T cell sensitizing signal-transducing peptide, a linker, and a translocation peptide; (ii) a T cell-sensitizing signal-transducing peptide; and (iii) a translocation peptide of 34-112 amino acid residues in length.
    Type: Grant
    Filed: December 3, 2013
    Date of Patent: November 1, 2016
    Assignee: TheVax Genetics Vaccine Co., Ltd.
    Inventors: Chia-Mao Wu, Hsiu-Kang Chang
  • Patent number: 9422559
    Abstract: This invention generally relates to a design and method for developing novel anti-tumor and/or anti-cancer drugs, vaccines and therapies, using microRNA and/or its shRNA homologs/mimics/derivatives. More specifically, the present invention relates to an use of a prokaryote-produced miRNA precursor (pro-miRNA) composition capable of being delivered into human cells and processed by the cells into mature miRNA effectors to elicit specific silencing effects on mir-302-targeted genes, subsequently leading to a beneficial result of tumor suppression and cancer therapy. The prokaryotic cells do not naturally express or process eukaryotic miRNA precursors (pre-miRNA); meanwhile, the present invention also teaches an inducible method for expressing pre-miRNAs, particularly mir-302 precursors by using the prokaryotic transcription system.
    Type: Grant
    Filed: August 12, 2013
    Date of Patent: August 23, 2016
    Inventors: Shi-Lung Lin, David T S Wu
  • Patent number: 9346869
    Abstract: Methods and compositions for treating obesity and related disorders. The methods include the use of stem cells treated with BMP-2, -4, -5, -6 and/or -7.
    Type: Grant
    Filed: March 19, 2014
    Date of Patent: May 24, 2016
    Assignee: Joslin Diabetes Center, Inc.
    Inventors: Yu-Hua Tseng, C. Ronald Kahn
  • Patent number: 9249426
    Abstract: The present invention concerns a pseudotyped viral vector particle for transferring biological material into cells, wherein said vector particle comprises at least:—a chimeric envelope glycoprotein which comprises or consists in a fusion of the transmembrane and extracellular domain of a baboon endogenous retrovirus (BaEV) envelope glycoprotein and the cytoplasmic tail domain of a murine leukemia virus (MLV) envelope glycoprotein; or—a modified BaEV envelope glycoprotein wherein the cytoplasmic tail domain is devoid of the fusion inhibitory R peptide.
    Type: Grant
    Filed: September 28, 2012
    Date of Patent: February 2, 2016
    Assignees: Institut National de la Sante et de la Recherche Medicale (INSERM), Ecole Normale Superieur de Lyon
    Inventors: Anais Girard-Gagnepain, Els Verhoeyen, Dimitri Lavillette, Francois-Loic Cosset
  • Patent number: 9192631
    Abstract: A method for obtaining human microglial precursor cells, comprising: (a) providing a cell population comprising neural precursor cells, wherein the cell population is obtainable from embryoid bodies differentiated from human pluripotent stem cells; (b) differentiating the cell population comprising neural precursor cells into microglial precursor cells by culturing in medium comprising a growth factor selected from the group consisting of insulin and insulin-like growth factors; (c) expanding and enriching microglial precursor cells in medium comprising a growth factor selected from the group consisting of insulin and insulin-like growth factors and 10 to 150 ng/ml GM-CSF; and (d) isolating microglial precursor cells comprising CD45-positive cells.
    Type: Grant
    Filed: April 28, 2010
    Date of Patent: November 24, 2015
    Assignee: Life & Brain GMBH
    Inventors: Harald Neumann, Kristin Roy, Oliver Brüstle, Michael Peitz
  • Patent number: 9169294
    Abstract: The disclosure is generally directed toward the design and synthesis of peptide amphiphile (PA) molecules that comprises a peptide configured to inhibit angiogenesis. The peptide amphiphile comprises a hydrophobic tail, peptide sequence capable of beta-sheet formation; and a peptide, wherein the peptide is configured to inhibit angiogenesis. Optionally the PA further comprises a flexible linker between the peptide sequence capable of beta-sheet formation and the peptides. Further this disclosure is directed to nanostructures comprising peptide amphiphiles configured to inhibit angiogenesis.
    Type: Grant
    Filed: March 10, 2014
    Date of Patent: October 27, 2015
    Assignee: NORTHWESTERN UNIVERSITY
    Inventors: Runye H. Zha, Ming Zhang, Samuel I. Stupp
  • Patent number: 9127248
    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: Grant
    Filed: June 28, 2013
    Date of Patent: September 8, 2015
    Assignee: Factor Bioscience Inc.
    Inventors: Matthew Angel, Christopher Rohde
  • Patent number: 9107910
    Abstract: Isolated liver progenitor stem cells and cell populations of isolated liver progenitor stem cells are disclosed. The progenitor stem cells originate from adult liver, especially human adult liver. The isolated progenitor stem cells have uses in medicine, hepatology, inborn errors of liver metabolism transplantation, infectious diseases and liver failure. Methods of isolating these cells and their culture is described. The isolated cells are characterized before and after differentiation. Their use for transplantation and as animal models of human disease, toxicology and pharmacology is disclosed.
    Type: Grant
    Filed: June 3, 2014
    Date of Patent: August 18, 2015
    Assignee: UNIVERSITÉ CATHOLIQUE DE LOUVAIN
    Inventors: Etienne Sokal, Mustapha Najimi
  • Patent number: 9109014
    Abstract: Provided herein is a nucleic acid comprising consensus amino acid sequence of foot-and-mouth disease FMDV VP1-4 coat proteins of FMDV subtypes A, Asia 1, C, O, SAT1, SAT2, and SAT3 as well as plasmids and vaccines expressing the sequences. Also provided herein is methods for generating an immune response against one or more FMDV subtypes using the vaccine as described above as well as methods for deciphering between vaccinated mammals with the vaccine and those that are infected with FMDV.
    Type: Grant
    Filed: November 2, 2010
    Date of Patent: August 18, 2015
    Assignees: The Trustees of the University of Pennsylvania, Inovio Pharmaceuticals, Inc.
    Inventors: David B Weiner, Bernadette Ferraro, Jian Yan, Patricia A Brown, Rodney A Bowling, Douglas R Kern, Mathura P Ramanathan, Niranjan Y Sardesai, Karuppiah Muthumani
  • Patent number: 9102796
    Abstract: The present invention relates to a polyalkyleneimine polymer having a repeat unit comprising a hydrophilic group, agents containing the same and methods for their use.
    Type: Grant
    Filed: December 12, 2008
    Date of Patent: August 11, 2015
    Assignee: Thermo Fisher Scientific Baltics UAB
    Inventors: Arunas Lagunavicius, Sarunas Zigmantas, Laurynas Riauba, Lolita Zaliauskiene, Richard Makuska, Ausvydas Vareikis, Ula Bernadisiute
  • Patent number: 9102949
    Abstract: The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.
    Type: Grant
    Filed: April 22, 2011
    Date of Patent: August 11, 2015
    Assignee: University of Massachusetts
    Inventors: Guangping Gao, Hongwei Zhang, Hongyan Wang, Zuoshang Xu
  • Patent number: 9096868
    Abstract: This invention provides a new approach to the design of a virus with a defective replication cycle, which can be rescued by wild type virus co-infection, and which expresses foreign antigenic epitopes that contribute to the elimination of virus infected cells and then to viral clearance. The vector of the invention, by expression of epitopes derived from common pathogens, by-passes existing tolerance of virus specific T cell responses. The vector will only replicate in virus infected cells.
    Type: Grant
    Filed: September 12, 2008
    Date of Patent: August 4, 2015
    Assignees: INSTITUT PASTEUR, INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
    Inventors: Qiang Deng, Marie-Louise Michel
  • Patent number: 9068174
    Abstract: Polynucleotides encoding a mutant human carboxylesterase enzyme and polypeptides encoded by the polynucleotides which are capable of metabolizing a prodrug and inactive metabolites thereof to active drug are provided. Compositions and methods for sensitizing cells to a prodrug agent, inhibiting cell growth, treating drug addiction, and facilitating the metabolism of an organophosphate with this enzyme are also provided. In addition, a screening assay for identification of drugs activated by this enzyme is described.
    Type: Grant
    Filed: January 24, 2012
    Date of Patent: June 30, 2015
    Assignees: St. Jude Children's Research Hospital, The University of North Carolina
    Inventors: Philip M. Potter, Monika Weirdl, Matthew R. Redinbo
  • Patent number: 9051584
    Abstract: A heat-resistant NDV live vaccine vector system includes a transcription plasmid, three helper plasmids, and host cells. The transcription plasmid is constructed by through cloning complete genomic cDNA of a heat-resistant NDV vaccine strain to a pBR322 vector. The three helper plasmids are constructed by cloning sequences coding nucleoprotein (NP), phosphoprotein, large polymerase protein of a heat-resistant NDV vaccine strain respectively to pcDNA3.1 vectors. A recombinant NDV artificially obtained by cotransfacting host cells with the transcription plasmid and the three helper plasmids shows heat-resistance.
    Type: Grant
    Filed: June 24, 2013
    Date of Patent: June 9, 2015
    Assignee: INSTITUTE OF ANIMAL HUSBANDRY AND VETERINARY SCIENCES, HUBEI ACADEMY OF AGRICULTURAL SCIENCES
    Inventors: Guoyuan Wen, Huabin Shao, Jun Yang, Honglin Wang, Qingping Luo, Rongrong Zhang, Diyun Ai, Ling Luo, Yu Shang, Jing Guo, Chen Chen
  • Publication number: 20150140663
    Abstract: The invention provides compositions of matter comprising a cowpea chlorotic mottle virus capsid protein (CCMV CP) and a ribonucleic acid, as well as methods for using such compositions. In such compositions, the cowpea chlorotic mottle virus capsid protein envelops the ribonucleic acid so as to for a capsid that can inhibit the degradation of the ribonucleic acid (e.g. by RNAses). A method of delivering a ribonucleic acid into the cytoplasm of a mammalian cell is also provided. Typically, the method comprises the steps of combining the mammalian cell with a composition of matter described herein under conditions selected to allow the cowpea chlorotic mottle virus capsid to contact the mammalian cell and deliver the ribonucleic acid into the cytoplasm of a mammalian cell.
    Type: Application
    Filed: November 13, 2014
    Publication date: May 21, 2015
    Inventors: William M. Gelbart, Charles M. Knobler, Rees F. Garmann, Odisse Azizgolshani, Ruben D. Cadena-Nava
  • Publication number: 20150133530
    Abstract: The present invention provides AAV capsid proteins comprising modification of one or a combination of the surface-exposed lysine, serine, threonine and/or tyrosine residues in the VP3 region. Also provided are rAAV virions comprising the AAV capsid proteins of the present invention, as well as nucleic acid molecules and rAAV vectors encoding the AAV capsid proteins of the present invention. Advantageously, the rAAV vectors and virions of the present invention have improved efficiency in transduction of a variety of cells, tissues and organs of interest, when compared to wild-type rAAV vectors and virions.
    Type: Application
    Filed: May 15, 2013
    Publication date: May 14, 2015
    Applicant: University of Florida Research Foundation, Inc.
    Inventors: Arun Srivastava, George Vladimirovich Aslanidi, Kim M. Van Vliet, Mavis Agbandje-Mckenna
  • Patent number: 9029152
    Abstract: This invention relates to the field of biotechnology or genetic engineering. Specifically, this invention relates to the field of gene expression. More specifically, this invention relates to novel substitution mutant receptors and their use in a Group H nuclear receptor-based inducible gene expression system and methods of modulating the expression of a gene in a host cell for applications such as gene therapy, large scale production of proteins and antibodies, cell-based high throughput screening assays, functional genomics and regulation of traits in transgenic organisms.
    Type: Grant
    Filed: September 13, 2012
    Date of Patent: May 12, 2015
    Assignee: RheoGene, Inc.
    Inventors: Subba Reddy Palli, Mohan Basavaraju Kumar, Dean Ervin Cress, Ted Tsutomu Fujimoto
  • Patent number: 9029142
    Abstract: Gene encoding human glucokinase mutant is provided. The gene has the nucleotide sequence chosen from the nucleotide sequence listed as SEQ ID NO:2 and the nucleotide sequence wherein the ORF region encodes the same amino acid sequence as the one encoded by ORF region (position 487 to 1884) of SEQ ID NO:2 and the rest of the region is same as the non-ORF region of SEQ ID NO:2. Human glucokinase mutant encoded by the gene, the recombinant vectors carrying the gene, the hosts comprising the vectors, pharmaceutical compositions thereof, uses thereof, and methods for treating and preventing diseases by using the same are provided. The human glucokinase mutant encoded by the gene has higher activity than that of the wild type human glucokinase, and thus provides a new way of controlling blood glucose and/or preventing and/or treating disturbance of carbohydrate metabolism, especially preventing and treating diabetes.
    Type: Grant
    Filed: July 7, 2011
    Date of Patent: May 12, 2015
    Inventor: Haidong Huang
  • Patent number: 9023648
    Abstract: The invention is directed to a chimeric gammaretrovirus comprising an gammaretroviral virion which contains a lentiviral Vpx protein and methods of use thereof. In a particular aspect, the chimeric gammaretrovirus is a chimeric murine leukemia virus (MLV) comprising an MLV virion which contains a lentiviral Vpx protein. The invention is also directed to use of the chimeric gammaretrovirus to produce a MLV that can transduce a non-dividing cell (G1/S/G2), transduce a non-dividing cell; enhance the ability of a MLV to transduce a non-dividing cell; transduce a quiescent (G0) cell; and enhance the ability of a human immunodeficiency virus 1 (HIV-1) to transduce a quiescent (G0) cell.
    Type: Grant
    Filed: December 29, 2010
    Date of Patent: May 5, 2015
    Assignee: University of Massachusetts
    Inventors: Mario Stevenson, Rajnish Kaushik, Xiaonan Zhu
  • Patent number: 9023646
    Abstract: The present invention provides HIV-derived lentivectors which are safe, highly efficient, and very potent for expressing transgenes for human gene therapy, especially, in human hematopoietic progenitor cells as well as in all other blood cell derivatives. The lentiviral vectors comprise a self-inactivating configuration for biosafety and promoters such as the EF1 ? promoter as one example. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element. These vectors therefore provide useful tools for genetic treatments such as inherited and acquired lympho-hematological disorders, gene-therapies for cancers especially the hematological cancers, as well as for the study of hematopoiesis via lentivector-mediated modification of human HSCs.
    Type: Grant
    Filed: September 9, 2013
    Date of Patent: May 5, 2015
    Assignee: Research Development Foundation
    Inventors: Didier Trono, Patrick Salmon
  • Publication number: 20150118201
    Abstract: The present invention provides methods of achieving directed evolution of viruses by in vivo screening or “panning” to identify viruses comprising scrambled AAV capsids having characteristics of interest, e.g., tropism profile and/or neutralization profile (e.g., ability to evade neutralizing antibodies). The invention also provides scrambled AAV capsids and virus particles comprising the same.
    Type: Application
    Filed: October 22, 2014
    Publication date: April 30, 2015
    Inventors: Xiao Xiao, Lin Yang
  • Patent number: 9017672
    Abstract: The invention provides a hexon Tat-PTD modified adenovirus, a gene delivery vector based on the modified adenovirus that enhances gene delivery efficiency, and an oncolytic agent based on the modified adenovirus that enhances tumor cell killing efficiency and improves therapeutic outcome.
    Type: Grant
    Filed: May 13, 2013
    Date of Patent: April 28, 2015
    Assignee: Immunicum Aktiebolag
    Inventors: Di Yu, Magnus Essand
  • Publication number: 20150111955
    Abstract: The invention relates to adeno-associated virus (AAV) serotype AAV-Rh74 and related AAV vectors, and AAV-Rh74 and related AAV vector mediated gene transfer methods and uses. In particular, AAV-Rh74 targets polynucleotides to cells, tissues or organs for expression (transcription) of genes encoding therapeutic proteins and peptides, and polynucleotides that function as or are transcribed into inhibitory nucleic acid sequences.
    Type: Application
    Filed: February 19, 2013
    Publication date: April 23, 2015
    Inventors: Katherine A. High, Federico Mingozzi, Junwei Sun, Philip Johnson
  • Patent number: 9012223
    Abstract: The disclosure provides methods for increasing genome stability of an embryonic stem (ES) cell or induced pluripotent stem (iPS) cell, increasing telomere length in an ES or iPS cell, or both, for example by contacting an ES or iPS cell with an agent that increases expression of Zscan4 in the cell. Methods for increasing genome stability or increasing telomere length in a population of ES or iPS cells are provided, for example by selecting Zscan4+ ES or iPS cells from the population of ES or iPS cells (which can include both Zscan4+ and Zscan4? ES or iPS cells). Therapeutic methods of using ES or iPS cells expressing Zscan4 are also provided. Further provided are methods of treating cancer by administering a Zscan4 polynucleotide or Zscan4 polypeptide. Also provided are methods of inducing differentiation of isolated ES or iPS cells into germ cells.
    Type: Grant
    Filed: April 23, 2014
    Date of Patent: April 21, 2015
    Assignee: The United States of America, as represented by the Secretary, Department of Health and Human Services
    Inventors: Minoru S. H. Ko, Michal Zalzman, Lioudmila V. Sharova
  • Patent number: 9012224
    Abstract: The present invention is based, in part, on the discovery that parvovirus (including AAV) capsids can be engineered to incorporate small, selective regions from other parvoviruses that confer desirable properties. The inventors have discovered that in some cases as little as a single amino acid insertion or substitution from a first parvovirus (e.g., an AAV) into the capsid structure of another parvovirus (e.g., an AAV) to create a chimeric parvovirus is sufficient to confer one or more of the desirable properties of the first parvovirus to the resulting chimeric parvovirus and/or to confer a property that is not exhibited by the first parvovirus or is present to a lesser extent.
    Type: Grant
    Filed: January 3, 2011
    Date of Patent: April 21, 2015
    Assignees: The University of North Carolina at Chapel Hill, The University of Florida Research Foundation
    Inventors: Dawn E. Bowles, Chengwen Li, Joseph E. Rabinowitz, Josh Grieger, Mavis Agbandje-McKenna, Richard Jude Samulski
  • Publication number: 20150105286
    Abstract: In this invention, a novel protein interaction domain is provided along with several of its variants. This domain is involved in protein-protein interactions with the Bcl-2 family of proteins. It is named BLID (Bcl2 family of proteins Like Interaction Domain). Several BLID peptides that could be useful for discovery of drugs to help fight pathological states like cancer are presented.
    Type: Application
    Filed: October 7, 2014
    Publication date: April 16, 2015
    Inventor: Carlos Witte-Hoffmann
  • Publication number: 20150093831
    Abstract: The embodiments disclosed herein relate to the construction of fully-deleted Adenovirus-based gene delivery vectors packaged without helper Adenovirus, and more particularly to their use in gene therapy for gene and protein expression, vaccine development, and immunosuppressive therapy for allogeneic transplantation. In an embodiment, a method for propagating an adenoviral vector includes (a) providing an Adenovirus packaging cell line; (b) transfecting a fully-deleted Adenoviral vector construct into the cell line; and optionally (c) transfecting a packaging construct into the cell line, wherein the fully-deleted Adenoviral vector construct and optionally the packaging construct can transfect the Adenovirus packaging cell line resulting in the encapsidation of a fully-deleted Adenoviral vector independent of helper Adenovirus. In an embodiment, a target cell is transduced with the encapsidated fully-deleted Adenoviral vector for treating a condition, disease or a disorder.
    Type: Application
    Filed: September 24, 2014
    Publication date: April 2, 2015
    Inventors: Miles B. Brennan, Erin K. Spiegel, Uwe D. Staerz, Charles Wall, Janae Wheeler, William J. Maslanik, Xianghua Zhang