Mouse (i.e., Mus) Patents (Class 435/354)
  • Patent number: 10034916
    Abstract: The present invention relates to a retinal pigment epithelial stem cell isolated from a posterior region of the retinal pigment epithelium of an adult mammal. The invention also relates to a method of inducing differentiation of retinal epithelial stem and progenitor cells in vitro, wherein the cells of the invention are highly plastic, multipotential stem cells. The invention also includes methods for the treatment of retinal diseases and vision loss involving the transplantation of retinal pigment epithelial stem cells or cells differentiated from retinal pigment epithelial stem cells to the retina of a patient in need of treatment.
    Type: Grant
    Filed: June 12, 2013
    Date of Patent: July 31, 2018
    Assignee: Regenerative Research Foundation
    Inventors: Sally Temple, Jeffrey Stern, Enrique L. Salero-Coca
  • Patent number: 9999207
    Abstract: A genetically modified mouse is provided that comprises a conditional Acvr1 allele that comprises a mutated exon that, upon induction, converts to a mutant exon phenotype, wherein the mutant exon phenotype includes ectopic bone formation. Mice comprising a mutant Acvr1 exon 5 in antisense orientation, flanked by site-specific recombinase recognition sites, are provided, wherein the mice further comprise a site-specific recombinase that recognizes the site-specific recombinase recognitions sites, wherein the recombinase is induced upon exposure of the mouse to tamoxifen. Upon exposure to tamoxifen, the recombinase is expressed and acts on the RRS-flanked mutant exon 5 and places the mutant exon 5 in sense orientation and deletes the wild-type exon.
    Type: Grant
    Filed: October 27, 2016
    Date of Patent: June 19, 2018
    Assignee: REGENERON PHARMACEUTICALS, INC.
    Inventors: Aris N. Economides, Sarah Jane Hatsell
  • Patent number: 9982232
    Abstract: The present invention relates to methods for generating mammalian multilineage-potential cells, including mesenchymal stem cells, comprising contacting mammalian somatic cells exhibiting a mature phenotype with PDGF-AB or functional derivative, fragment or mimetic thereof and Azacitidine or functional derivative or analog thereof for a time and under conditions sufficient to induce the transition of the somatic cells to cells exhibiting multilineage differentiative potential. Also provided are uses of said multilineage-potential cells, such as in promoting tissue repair and regeneration.
    Type: Grant
    Filed: December 17, 2013
    Date of Patent: May 29, 2018
    Inventors: John Pimanda, Vashe Chandrakanthan
  • Patent number: 9745549
    Abstract: [Problem] To provide a cell culture substrate, and a cell culturing method using the substrate and a method for inducing differentiation of pluripotent stem cells using the substrate, which allow culturing of pluripotent stem cells and allow differentiation of pluripotent stem cells into a specified cell species, particularly neural and neural progenitor cells, at a high purity. [Means for Solution] A cell culture substrate, characterized in that, onto the surface, one or more selected from the group consisting of N-cadherin, a fusion protein comprising an entire or partial region of N-cadherin, and a fusion protein comprising an entire or partial region of a protein homologous to N-cadherin are immobilized or coated.
    Type: Grant
    Filed: April 18, 2013
    Date of Patent: August 29, 2017
    Assignees: SOMAR CORP.
    Inventors: Amranul Haque, Masato Nagaoka, Toshihiro Akaike
  • Patent number: 9695442
    Abstract: Disclosed herein are methods and compositions for targeted deletion of double-stranded DNA. The compositions include fusion proteins comprising a cleavage domain (or cleavage half-domain) and an engineered zinc finger domain, and polynucleotides encoding same. Methods for targeted deletion include introduction of such fusion proteins, or polynucleotides encoding same, into a cell such that two targeted cleavage events occur. Subsequent cellular repair mechanisms result in deletion of sequences between the two cleavage sites.
    Type: Grant
    Filed: March 4, 2013
    Date of Patent: July 4, 2017
    Assignee: Sangamo Therapeutics, Inc.
    Inventors: Dmitry Guschin, Fyodor Urnov
  • Patent number: 9657274
    Abstract: The present invention provides for methods, compositions, and kits for producing an induced pluripotent stem cell from a non-pluripotent mammalian cell using a 3?-phosphoinositide-dependent kinase-1 (PDK1) activator or a compound that promotes glycolytic metabolism as well as other small molecules.
    Type: Grant
    Filed: March 14, 2016
    Date of Patent: May 23, 2017
    Assignee: The Scripps Research Institute
    Inventors: Saiyong Zhu, Sheng Ding
  • Publication number: 20150150152
    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 a novel ecdysone receptor/chimeric retinoid X receptor-based inducible gene expression system and methods of modulating gene expression 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: Application
    Filed: November 24, 2014
    Publication date: May 28, 2015
    Inventors: Marianna Zinovievna KAPITSKAYA, Subba Reddy Palli
  • Publication number: 20150143561
    Abstract: Non-human animals, cells, methods and compositions for making and using the same are provided, wherein the non-human animals and cells comprise a humanized B-cell activating factor gene. Non-human animals and cells that express a human or humanized B-cell activating factor protein from an endogenous B-cell activating factor locus are described.
    Type: Application
    Filed: December 5, 2014
    Publication date: May 21, 2015
    Inventors: John McWhirter, Cagan Gurer, Lynn Macdonald, Andrew J. Murphy
  • Publication number: 20150141289
    Abstract: The invention provides for compositions and methods for identifying and validating modulators of cell fate, such as such as maintenance, cell specification, cell determination, induction of stem cell fate, cell differentiation, cell dedifferentiation, and cell trans-differentiation. The invention relates to reporter nucleic acid constructs, host cells comprising such constructs, and methods using such cells and constructs. The invention relates to methods for making cells comprising one or more reporter nucleic acid constructs using fluorogenic oligonucleotides. The methods relate to high throughput screens.
    Type: Application
    Filed: January 30, 2015
    Publication date: May 21, 2015
    Inventors: Kambiz Shekdar, Dennis J. Sawchuk, Jessica C. Langer
  • Publication number: 20150143562
    Abstract: Non-human animals, cells, methods and compositions for making and using the same are provided, wherein the non-human animals and cells comprise a humanized a proliferation-inducing ligand gene. Non-human animals and cells that express a human or humanized a proliferation-inducing ligand protein from an endogenous a proliferation-inducing ligand locus are described.
    Type: Application
    Filed: December 5, 2014
    Publication date: May 21, 2015
    Inventors: John McWhirter, Cagan Gurer, Lynn Macdonald, Andrew J. Murphy
  • Publication number: 20150143558
    Abstract: Non-human animals, cells, methods and compositions for making and using the same are provided, wherein the non-human animals and cells comprise a humanized B-cell activating factor gene. Non-human animals and cells that express a human or humanized B-cell activating factor protein from an endogenous B-cell activating factor locus are described.
    Type: Application
    Filed: November 10, 2014
    Publication date: May 21, 2015
    Inventors: John McWhirter, Cagan Gurer, Lynn Macdonald, Andrew J. Murphy
  • Patent number: 9034649
    Abstract: This invention relates to a method for producing a protein of interest, comprising introducing a protein expression vector which comprises a gene fragment a gene fragment comprising a DNA encoding a protein of interest and a selectable marker gene and transposon sequences at both terminals of the gene fragment, into a suspension mammalian cell; integrating the gene fragment inserted between a pair of the transposon sequences, into a chromosome of the mammalian cell to obtain a mammalian cell capable of expressing the protein of interest; and suspension-culturing the mammalian cell; and a suspension mammalian cell capable of expressing the protein of interest.
    Type: Grant
    Filed: June 11, 2010
    Date of Patent: May 19, 2015
    Assignees: Inter-University Research Institute Corporation Research Organization of Information and Systems, KYOWA HAKKO KIRIN CO., LTD
    Inventors: Koichi Kawakami, Keina Yamaguchi, Risa Ogawa, Masayoshi Tsukahara
  • Publication number: 20150132853
    Abstract: Methods for de-differentiating or altering the life-span of desired “recipient” cells, e.g., human somatic cells, by the introduction of cytoplasm from a more primitive, less differentiated cell type, e.g., oocyte or blastomere are provided. These methods can be used to produce embryonic stem cells and to increase the efficiency of gene therapy by allowing for desired cells to be subjected to multiple genetic modifications without becoming senescent. Such cytoplasm may be fractionated and/or subjected to subtractive hybridization and the active materials (sufficient for de-differentiation) identified and produced by recombinant methods.
    Type: Application
    Filed: June 11, 2014
    Publication date: May 14, 2015
    Applicant: Advanced Cell Technology, Inc.
    Inventor: Karen B. Chapman
  • Publication number: 20150132845
    Abstract: Devices, systems, and methods for continuous cell culture and other reactions are generally described. In some embodiments, chambers (e.g., cell growth chambers) including at least a portion of a wall formed of a flexible member are provided. A retaining structure can be incorporated outside and proximate to the chamber such that when liquid is added to the chamber, the flexible member is consistently and predictably deformed, and a consistent volume of liquid is added. The flexible member can be formed of, in some embodiments, a gas-permeable medium. In some embodiments, reaction chambers can be arranged in a fluidic loop, and a bypass channel can be used to introduce and/or extract fluid from the loop without affecting loop operation.
    Type: Application
    Filed: November 6, 2014
    Publication date: May 14, 2015
    Applicant: Massachusetts Institute of Technology
    Inventors: Rajeev Jagga Ram, Kevin Shao-Kwan Lee
  • Publication number: 20150133317
    Abstract: Disclosed herein are compositions and methods for sequencing, analyzing, and utilizing samples such as single samples. Also disclosed herein are compositions and methods for matching together two or more sequences from a sample. Also disclosed herein are compositions and methods for expressing and screening molecules of interest.
    Type: Application
    Filed: April 27, 2012
    Publication date: May 14, 2015
    Applicants: Department of Veterans Affairs, The Board of Trustees of the Leland Stanford Junior University
    Inventors: William H. Robinson, Yann Chong Tan, Jeremy Sokolove
  • Publication number: 20150133531
    Abstract: The present invention provides a method of expressing at least one heterologous nucleic acid sequence in a cell, the method comprising introducing at least one heterologous nucleic acid sequence into a cell by infecting said cell with a recombinant negative-strand RNA virus vector comprising said at least one heterologous nucleic acid sequence, wherein the recombinant negative-strand RNA virus vector includes a viral genome coding for a mutated P protein, which leads to a loss of the viral genome replication ability without a loss of the viral transcription ability, and wherein said at least one heterologous nucleic acid sequence encodes a cellular reprogramming or programming factor or a therapeutic protein. In addition, the present invention provides a cell or a population of cells prepared in vitro by said method as well as a pharmaceutical composition comprising said cell or population of cells.
    Type: Application
    Filed: May 24, 2013
    Publication date: May 14, 2015
    Applicant: AmVac AG
    Inventor: Marian Wiegand
  • 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: 9029330
    Abstract: Modified interleukin-12 (IL-12) p40 polypeptides are disclosed. The modified polypeptides have alterations in the IL-12p40 subunit to eliminate the protease site between positions Lys260 and Arg261. The modified IL-12p40 polypeptides according to the invention have improved stability compared to wild-type mature human IL-12p40 polypeptides.
    Type: Grant
    Filed: May 23, 2012
    Date of Patent: May 12, 2015
    Assignee: Merck Patent GmbH
    Inventors: Gordon D. Webster, Suzanne P. McKenzie, Kin-Ming Lo, Pascal André Stein
  • Publication number: 20150125951
    Abstract: The present invention relates to a mutant human alpha-synuclein with increased toxicity compared to wild-type alpha-synuclein, or a homologue thereof, wherein the mutant alpha-synuclein or homologue thereof comprises at least one amino acid substitution selected from the group consisting of a substitution at the alanine at position 56 (A56), at the alanine at position 76 (A76), at the methionine at position 127 (M127) and/or at the valine at position 118 (V118), as defined in the claims. Further, the invention relates to a polynucleotide encoding the mutant alpha-synuclein or homologue thereof, or an expression vector comprising said polynucleotide, a cell comprising the polynucleotide or expression vector, as defined in the claims. Also, a non-human animal comprising the cell of the invention is provided, as defined in the claims. Finally, the invention provides methods for identifying a substance that prevents or reduces toxicity of alpha-synuclein, as defined in the claims.
    Type: Application
    Filed: July 8, 2014
    Publication date: May 7, 2015
    Inventors: Markus ZWECKSTETTER, Pinar KARPINAR, Christian GRIESINGER
  • Publication number: 20150118747
    Abstract: An in vitro model system that guides the development of microvasculature, recapitulating the detailed organization of both its cellular and a-cellular components is established. Use of electrostretched fibrin microfibers enables both endothelial layer organization and co-culture of supporting perivascular (mural) cells such as vascular smooth muscle cells and pericytes. The fiber curvature affects the circumferential deposition of endothelial-produced ECM independently of cellular organization and induces deposition of higher quantities of vascular ECM proteins. Further, a luminal multicellular microvascular structure is disclosed.
    Type: Application
    Filed: October 31, 2014
    Publication date: April 30, 2015
    Inventors: Sharon Gerecht, Shuming Zhang, Sebastian F. Barreto Ortiz, Hai-Quan Mao
  • 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: 9005962
    Abstract: The present invention relates to the generation of anterior definitive endoderm (ADE) cells from embryonic stem cells and the differentiation of such cells to, for example, pancreatic or liver cells. The invention also relates to cell lines, cell culture methods, cells markers and the like and their potential uses in a variety of applications.
    Type: Grant
    Filed: August 25, 2008
    Date of Patent: April 14, 2015
    Assignee: The University Court of the University of Edinburgh
    Inventors: Gillian Mary Morrison, Joshua Mark Brickman, Ifigenia Oikonomopoulou
  • Patent number: 8993330
    Abstract: A composition of a female germinal cell (egg) extract of pluricellular organisms in M-phase of the cell cycle, the extract being used for a mitotic remodeling of chromosomes of donor cells of pluricellular organisms, wherein the mitotic remodeling confers to the nucleus of the donor cells the ability to adapt themselves to the early embryonic development, in particular to the replication phases, in order to carry out the embryonic development or to obtain stem cells.
    Type: Grant
    Filed: May 5, 2014
    Date of Patent: March 31, 2015
    Assignee: Centre National de la Recherche Scientifique
    Inventors: Marcel Mechali, Jean-Marc Lemaitre
  • Patent number: 8993328
    Abstract: The present invention provides systems and methods for improving the efficiency of a transient gene delivery system to differentiating embryonic stem (ES) cells by serum starving the targeted cells for one to three days prior to transfection. Such a serum starvation surprisingly resulted in increased expression of a constitutively-controlled plasmid from 50.4% to 83.2% of the population and increased expression of a promoter/enhancer controlled plasmid from ˜1.4% to ˜3.7% of the population.
    Type: Grant
    Filed: September 22, 2009
    Date of Patent: March 31, 2015
    Assignee: Rutgers, The State University of New Jersey
    Inventors: Martin L. Yarmush, Eric J. Wallenstein, Rene S. Schloss
  • Publication number: 20150089680
    Abstract: Genetically modified mice are provided that express human ? variable (hV?) sequences, including mice that express hV? sequences from an endogenous mouse ? light chain locus, mice that express hV? sequences from an endogenous mouse ? light chain locus, and mice that express hV? sequences from a transgene or an episome wherein the hV? sequence is linked to a mouse constant sequence. Mice are provided that are a source of somatically mutated human ? variable sequences useful for making antigen-binding proteins. Compositions and methods for making antigen-binding proteins that comprise human ? variable sequences, including human antibodies, are provided.
    Type: Application
    Filed: December 5, 2014
    Publication date: March 26, 2015
    Inventors: Lynn Macdonald, Sean Stevens, Cagan Gurer, Andrew J. Murphy, Karolina A. Meagher
  • Publication number: 20150089679
    Abstract: Genetically modified non-human animals and methods and compositions for making and using the same are provided, wherein the genetic modification comprises a humanization of an endogenous signal-regulatory protein gene, in particular a humanization of a SIRP? gene. Genetically modified mice are described, including mice that express a human or humanized SIRP? protein from an endogenous SIRP? locus.
    Type: Application
    Filed: October 17, 2014
    Publication date: March 26, 2015
    Applicant: REGENERON PHARMACEUTICALS, INC.
    Inventors: Andrew J. Murphy, O. Gavin Thurston, Bindu Varghese, Cagan Gurer
  • Publication number: 20150079634
    Abstract: The present invention is related to a method to reduce peptide amidation activity in a given cell line, cell lines with reduced peptide amidation activity, and uses thereof.
    Type: Application
    Filed: April 16, 2013
    Publication date: March 19, 2015
    Inventors: Mihaela Skulj, Dominik Gaser
  • Publication number: 20150071883
    Abstract: The invention provides improved adeno-associated virus (AAV) Factor VIII (FVIII) vectors, including AAV FVIII vectors that produce a functional Factor VIII polypeptide and AAV FVIII vectors with high expression activity.
    Type: Application
    Filed: September 10, 2014
    Publication date: March 12, 2015
    Inventor: Peter Cameron Colosi
  • Publication number: 20150072353
    Abstract: Analyte sensors, methods for producing and using analyte sensors, methods of detecting and/or measuring analyte activity, detecting pH change, and/or, controlling the concentration of an analyte in a system, are disclosed. Embodiments of the analyte sensors according to the disclosure can provide an accurate and convenient method for characterizing analyte activity, detecting pH change, controlling the concentration of an analyte in a system, and the like, in both in vivo and in vitro environments, in particular in living cell imaging.
    Type: Application
    Filed: August 21, 2014
    Publication date: March 12, 2015
    Inventors: Jenny Jie Yang, Shen Tang
  • Patent number: 8975068
    Abstract: Disclosed herein are methods for controlling stem cell differentiation through the introduction of transgenes having Xic, Tsix, Xite, or Xic flanking region sequences to block differentiation and the removal of the transgenes to allow differentiation. Also disclosed are small RNA molecules and methods for using the small RNA molecules to control stem cell differentiation. Also disclosed are stem cells genetically modified by the introduction of Xic, Tsix, XUe, or Xic flanking region sequences.
    Type: Grant
    Filed: January 24, 2008
    Date of Patent: March 10, 2015
    Assignee: The General Hospital Corporation
    Inventor: Jeannie T. Lee
  • Publication number: 20150065560
    Abstract: The present invention relates to a one-vector expression system comprising a sequence encoding two polypeptides, such as tyrosine hydroxylase (TH) and GTP-cyclohydrolase 1 (GCH1). The two polypeptides can be should preferentially be expressed at a ratio between 3:1 and 15:1, such as between 3:1 and 7:1. The invention is useful in the treatment of catecholamine deficient disorders, such as dopamine deficient disorders including but not limited to Parkinson's Disease. Moreover, the present invention provides a method to deliver the vector construct in order to limit the increased production of the catecholamine to the cells in need thereof.
    Type: Application
    Filed: April 1, 2014
    Publication date: March 5, 2015
    Inventors: Tomas Björklund, Anders Björklund, Deniz Kirik
  • Publication number: 20150064148
    Abstract: The present invention provides a method for improving pancreatic function in a subject in need thereof, the method comprising administering to the subject STRO-1+ cells and/or progeny cells thereof and/or soluble factors derived therefrom. The method of the invention is useful for treating and/or preventing and/or delaying the onset or progression of a disorder resulting from or associated with pancreatic dysfunction, e.g., resulting from abnormal endocrine or exocrine function of the pancreas.
    Type: Application
    Filed: November 7, 2014
    Publication date: March 5, 2015
    Applicant: MESOBLAST, INC.
    Inventors: Silviu Itescu, Ravi Krishnan
  • Patent number: 8961955
    Abstract: Various embodiments of the present invention include compositions, materials and methods for maintaining and propagating mammalian mesenchymal stem cells in an undifferentiated state in the absence of feeder cells and applications of the same.
    Type: Grant
    Filed: February 8, 2013
    Date of Patent: February 24, 2015
    Assignees: The Board of Trustees of the University of Arkansas, The United States of America as Represented by the Department of Veterans Affair
    Inventors: Xiao-Dong Chen, Robert L. Jilka
  • Publication number: 20150052625
    Abstract: The present invention provides embryonic stem cells obtainable from an embryo of an immunodeficient mouse which is deficient in both Rag2 and Jak3 genes by culture in the presence of a GSK3 inhibitor and an MEK inhibitor, as well as a transgenic mouse, which is created with the use of these embryonic stem cells.
    Type: Application
    Filed: March 27, 2012
    Publication date: February 19, 2015
    Applicants: Trans Genic Inc., National University Corporation Kumamoto University
    Inventors: Ken-ichi Yamamura, Kimi Araki, Seiji Okada, Akihiko Shimono
  • Publication number: 20150050211
    Abstract: Provided herein are methods to generate and screen peptides that exhibit drug like stabilities in vitro and in vivo. By selecting for enzyme resistance, Applicants are able to derive peptides that are not only stable to a broad spectrum of proteases, but also stable to other drug processing enzymes such as cytochrome P450s. This approach provides a general method to the rapid development of highly stable peptides for therapeutic development and diagnosis. The peptides are further modified for oral bioavailability.
    Type: Application
    Filed: August 31, 2012
    Publication date: February 19, 2015
    Applicant: University of Souththern California
    Inventors: Stephen V. Fiacco, Terry T. Takahashi, Richard W. Roberts
  • Patent number: 8956873
    Abstract: The present invention is directed generally to eukaryotic host cells comprising artificial endosymbionts and methods of introducing artificial endosymbionts into eukaryotic host cells. The invention provides artificial endosymbionts that introduce a phenotype to host cells that is maintained in daughter cells. The invention additionally provides eukaryotic host cells containing magnetotactic bacteria.
    Type: Grant
    Filed: January 13, 2012
    Date of Patent: February 17, 2015
    Assignee: Bell Biosystems, Inc.
    Inventors: Caleb B. Bell, III, Alexey V. Bazarov
  • Publication number: 20150044768
    Abstract: The method of the invention provides for producing a heterologous protein in mammalian host cells having nucleic acid encoding Hepatitis B X protein and the heterologous protein, by growing mammalian host cells selected from the group consistng of HKB11, CHO, BHK21, C2C12, and HEK293 cells, by growing mammalian host cells in non-adherent suspension culture, or by growing mammalian host cells which contain nucleic acid providing exogenous X-box Binding Protein, XBP1s. The conditions should be such that HBx, exogenous XBP1s if present, and the heterologous protein are expressed by the mammalian cells. The invention includes compositions for carrying out the method.
    Type: Application
    Filed: October 24, 2014
    Publication date: February 12, 2015
    Inventors: FANG JIN, RICHARD N. HARKINS, MAXINE BAUZON, TERRY HERMISTON
  • Publication number: 20150044772
    Abstract: An inactive CRISPR/Cas system-based fusion protein and its applications in gene editing are disclosed. More particularly, chimeric fusion proteins including an inCas fused to a DNA modifying enzyme and methods of using the chimeric fusion proteins in gene editing are disclosed. The methods can be used to induce double-strand breaks and single-strand nicks in target DNAs, to generate gene disruptions, deletions, point mutations, gene replacements, insertions, inversions and other modifications of a genomic DNA within cells and organisms.
    Type: Application
    Filed: August 8, 2014
    Publication date: February 12, 2015
    Inventor: Guojun Zhao
  • Patent number: 8951799
    Abstract: The present disclosure is directed to the development of compositions, such as extracellular matrices, and processes for using the same, that both maintain stem cells in vitro pluripotency and enable self-renewal. In this regard, it has been discovered that when pluripotent mouse and human embryonic stem cells are cultured on plates coated with recombinant laminin-10 (laminin-511) or their functional domains, in the absence of differentiation inhibitors or feeder cells, the embryonic stem cells proliferated and maintained their pluripotency.
    Type: Grant
    Filed: August 25, 2009
    Date of Patent: February 10, 2015
    Assignee: BioLamina AB
    Inventors: Anna Domogatskaya, Sergey Rodin, Karl Tryggvason
  • Publication number: 20150037883
    Abstract: The various embodiments herein provide a method for derivation and long term establishment of ground state pluripotent embryonic stem cells. Further the embodiments herein provides a method to inhibit the ERK and TGF ? signalling pathways for long term maintenance of the embryonic stem cells. The R2i mouse embryonic stem (ES) cells are derived from 3.5 day blastocysts. The mouse ES cells are cultured in media containing R2i and 2i inhibitors of ERK and TGF ? pathways. The ES cells are subjected to in vitro and in vivo differentiation. The ES cells are subjected to RT-PCR and qRT-PCR, flow cytometry and karyotyping. The result reveals that the R2i maintains the ground state of ES cells and self renewal. Also R2i increases embryonic cleavage and clonal propagation of ES. Further R2i asserts genomic integrity and pluripotency of ES.
    Type: Application
    Filed: August 19, 2014
    Publication date: February 5, 2015
    Applicant: ROYAN INSTITUTE
    Inventors: Hossein Baharvand, Seyedeh Nafiseh Nafiseh Hasani, Mehdi Totonchi, Hamid Gourabi
  • Publication number: 20150037292
    Abstract: Stromal stem cells are prospectively isolated from human bone marrow then expanded into clonal populations and cultured and used, the isolation being on the basis of expression of a cell surface marker, wherein the cell surface marker binds an antibody and wherein said antibody cross reacts with a cell surface marker found on mouse stromal stem cells or rat stromal stem cells, and optionally also on a cell of at least one other mammalian species selected from mouse, rat, horse, rabbit and pig cells. Useful stromal stem cell populations are positive for SDC2.
    Type: Application
    Filed: February 11, 2013
    Publication date: February 5, 2015
    Applicant: Orbsen Therapeutics Limited
    Inventor: Stephen Joseph Elliman
  • Patent number: 8946504
    Abstract: Targeting constructs and methods of using them are provided for differentiation-dependent modification of nucleic acid sequences in cells and in non-human animals. Targeting constructs comprising a promoter operably linked to a recombinase are provided, wherein the promoter drives transcription of the recombinase in an differentiated cell but not an undifferentiated cell. Promoters include Blimp1, Prm1, Gata6, Gata4, Igf2, Lhx2, Lhx5, and Pax3. Targeting constructs with a cassette flanked on both sides by recombinase sites can be removed using a recombinase gene operably linked to a 3?-UTR that comprises a recognition site for an miRNA that is transcribed in undifferentiated cells but not in differentiated cells. The constructs may be included in targeting vectors, and can be used to automatically modify or excise a selection cassette from an ES cell, a non-human embryo, or a non-human animal.
    Type: Grant
    Filed: July 3, 2013
    Date of Patent: February 3, 2015
    Assignee: Regeneron Pharmaceuticals, Inc.
    Inventors: David Frendewey, Guochun Gong, Ka-Man Venus Lai, David M. Valenzuela
  • Patent number: 8940960
    Abstract: The human Occludin protein is identified as an essential Hepatitis C Virus (HCV) cell entry factor. Occludin is shown to render murine and other non-human cells infectable with HCV and to be required for HCV-susceptibility of human cells. Associated methods for inhibiting HCV infection, transgenic animal models for HCV pathogenesis, methods of identifying compounds or agents that prevent or mitigate interaction of HCV with Occludin, and HCV inhibitory agents are also disclosed. Kits and cell culture compositions useful for identifying compounds or agents that prevent or mitigate interaction of HCV with Occludin are also provided.
    Type: Grant
    Filed: October 1, 2009
    Date of Patent: January 27, 2015
    Assignee: The Rockefeller University
    Inventors: Alexander Ploss, Matthew Evans, Charles Rice
  • Publication number: 20150023995
    Abstract: The present invention provides polypeptides having a composite amino acid sequence derived from a consensus sequence representing the capsid proteins of two or more circulating strains of a non-enveloped virus. In particular, the invention provides virus-like particles comprising at least one composite polypeptide. Such virus-like particles have antigenic epitopes of two or more circulating strains of a non-enveloped virus and produce an increase in antisera cross-reactivity to one or more circulating strains of the non-enveloped virus. Methods of making composite virus-like particles and vaccine formulations comprising composite virus-like particles are also disclosed.
    Type: Application
    Filed: July 25, 2014
    Publication date: January 22, 2015
    Applicant: TAKEDA VACCINES, INC.
    Inventors: Charles RICHARDSON, Robert F. BARGATZE, Joel HAYNES, Bryan STEADMAN
  • Publication number: 20150026831
    Abstract: This invention relates transgenic animals that overexpress TL1A in a tissue specific manner to model inflammatory bowel disease (IBD), such as colitis, Crohn's disease and ulcerative colitis, fibrosis, and related inflammatory diseases and conditions. TL1A transgenic animals constitutively express both TL1A and GFP in lymphoid and myeloid cell lineages, allowing convenient identification and sorting of immune cells involved in IBD disease progression, such as T-cells, antigen presenting cells (APC), and dendritic cells (DC). TL1A transgenic animals may be induced to exhibit gross fibrosis, or isolated cells may be implanted into immunodeficient mice to establish colitis.
    Type: Application
    Filed: May 8, 2014
    Publication date: January 22, 2015
    Applicant: CEDARS-SINAI MEDICAL CENTER
    Inventors: David Q. Shih, Stephan R. Targan
  • Publication number: 20150026833
    Abstract: An obese mouse model was developed by overexpressing the mitochondrial protein prohibitin (PHB) in white adipose tissue (WAT) specific manner driven by adipocyte protein 2 (aP2) promoter. These mice begin to develop obesity as a result of mitochondrial remodeling (upregulation of mitochondrial biogenesis and function) in WAT.
    Type: Application
    Filed: July 11, 2014
    Publication date: January 22, 2015
    Inventors: Sudharsana R. Ande, Suresh Mishra
  • Publication number: 20150020223
    Abstract: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are delivery systems and tissues or organ which are targeted as sites for delivery. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.
    Type: Application
    Filed: September 9, 2014
    Publication date: January 15, 2015
    Inventors: Feng Zhang, Randall Jeffrey Platt, Guoping Feng, Yang Zhou
  • Publication number: 20150018522
    Abstract: Disclosed are mutants of galactosyltransferases that can catalyze formation of oligosaccharides in the presence of magnesium; mutants of galactosyltransferases having altered donor and acceptor specificity which can catalyze formation of oligosaccharides in the presence of magnesium; methods and compositions that can be used to synthesize oligosaccharides; methods for increasing the immunogenicity of an antigen; and methods to stabilize platelets.
    Type: Application
    Filed: March 10, 2014
    Publication date: January 15, 2015
    Applicant: The United States of America, as represented by the Secretary, Department of Health & Human Servic
    Inventors: Pradman K. Qasba, Elizabeth Boeggeman, Boopathy Ramakrishnan
  • Publication number: 20150010937
    Abstract: The present invention is directed generally to eukaryotic cells comprising single-celled organisms that are introduced into the eukaryotic cell through human intervention and which transfer to daughter cells of the eukaryotic cell, and methods of introducing such single-celled organisms into eukaryotic cells. The invention provides single-celled organisms that introduce a phenotype to eukaryotic cells that is maintained in daughter cells. The invention additionally provides eukaryotic cells containing magnetic bacteria. The invention further provides eukaryotic cells engineered with single-celled organisms to allow for multimodal observation of the eukaryotic cells. Each imaging method (or modality) allows the visualization of different aspects of anatomy and physiology, and combining these allows the imager to learn more about the subject being imaged.
    Type: Application
    Filed: July 15, 2014
    Publication date: January 8, 2015
    Inventors: Caleb B. Bell, III, Alexey Bazarov
  • Publication number: 20150005369
    Abstract: Disclosed are methods of gene delivery using capsid-modified recombinant adeno-associated viral (rAAV) vectors. Exemplary methods are provided employing vectors that have altered affinity for heparin or heparin sulfate, as well as vectors, expression systems, and rAAV virions that lack functional VP2 protein expression, but are nevertheless, fully virulent. Also provided by the invention are methods employing the rAAV vector-based compositions, virus particles, host cells, and pharmaceutical formulations in the expression of selected therapeutic proteins, polypeptides, peptides, antisense oligonucleotides and/or ribozymes in selected mammals, including organs, tissues, and human host cells.
    Type: Application
    Filed: July 8, 2014
    Publication date: January 1, 2015
    Inventors: Nicholas Muzyczka, Shaun R. Opie, Kenneth H. Warrington