Abstract: Eukaryotic protein-coding messenger RNAs and non-coding microRNAs are naturally transcribed by type II RNA polymerases (pol-2) but not prokaryotic RNA polymerases. As a result, current eukaryotic RNA and protein production is performed either using eukaryotic pol-2 promoters in hybridomas or mammalian cells or using prokaryotic promoters in bacterial cells. However, because prokaryotic RNA transcription tends to be error-prone, frequent mutation is a big problem. Also, growing hybridomas or mammalian cells is relatively laborious and costly. To overcome these problems, the present invention provides a novel inducible composition and method for producing eukaryotic RNAs and/or their related peptides/proteins directly using eukaryotic pol-2 promoter-driven gene expression in fast growing bacteria, without the need of changing to prokaryotic promoters or growing hybridomas/mammalian cells.
Abstract: Described herein are methods of preventing, arresting progression of or ameliorating vision loss and other conditions associated with retinitis pigmentosa and x-linked retinitis pigmentosa in a subject. The methods include administering to a subject an effective concentration of a composition comprising a recombinant adeno-associated virus (AAV) carrying a nucleic acid sequence encoding a normal retinitis pigmentosa GTPase regulator (RPGR gene), or fragment thereof, under the control of regulatory sequences which express the product of the gene in the photoreceptor cells of the subject, and a pharmaceutically acceptable carrier.
Type:
Grant
Filed:
January 23, 2013
Date of Patent:
September 26, 2017
Assignees:
The Trustees of the University of Pennsylvania, Univeristy of Florida Research Foundation, Incorporated
Inventors:
William A Beltran, Gustavo D Aguirre, Samuel G Jacobson, Artur V Cideciyan, Alfred S Lewin, Sanford L Boye, William W Hauswirth, Wen-Tao Deng
Abstract: An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes are alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.
Type:
Grant
Filed:
April 7, 2015
Date of Patent:
September 19, 2017
Assignees:
UCL BUSINESS PLC, THROMBOSIS RESEARCH INSTITUTE, ST. JUDE CHILDREN'S RESEARCH HOSPITAL
Inventors:
Amit Nathwani, Natalie Ward, Adrian Thrasher, Edward Tuddenham, John Mcvey, John Gray, Andrew Davidoff
Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.
Abstract: The present invention relates to methods and compositions for treating, ameliorating or preventing a disease or disorder in a subject by introducing into cells of the subject a therapeutic gene switch construct that controls expression of one or more therapeutic products.
Type:
Grant
Filed:
September 24, 2015
Date of Patent:
August 8, 2017
Assignee:
Intrexon Corporation
Inventors:
Bethany Lynn Merenick, Robert P. Beech, Thomas D. Reed, Anna P. Tretiakova, Richard E. Peterson
Abstract: Methods of generating sequence diversity in a protein, such as a ligand-binding protein, are provided. The methods comprise targeted introduction of two or more recombination signal sequences (RSSs) into the protein coding sequence and introduction of the modified protein coding sequence into a recombination-competent host cell, specifically a recombination-competent host cell that is capable of expressing at least RAG-1 and RAG-2, thereby allowing for recombination of the protein coding sequence and expression of variant proteins. Also provided are polynucleotides comprising a nucleic acid sequence encoding a target protein, such as a ligand-binding protein, and comprising two or more RSSs, and compositions and host cells comprising same.
Type:
Grant
Filed:
March 14, 2013
Date of Patent:
August 1, 2017
Assignee:
INNOVATIVE TARGETING SOLUTIONS INC.
Inventors:
Michael Gallo, Jaspal Singh Kang, Craig Robin Pigott, Abby Lin
Abstract: Methods for delivering a therapeutic agent to a cerebral cortex or to the cerebral cortex and spinal cord include administering a viral vector that includes the therapeutic agent into white matter of a brain in order to deliver the therapeutic agent to the cerebral cortex or to the cerebral cortex and spinal cord. Kits for performing such methods may include at least one viral vector, at least one therapeutic agent, and a convection enhanced delivery (CED) catheter.
Abstract: The present invention provides a rat embryonic stem cell characterized by having the following properties of (a) expressing Oct3/4 gene and Nanog gene, (b) positive for alkaline phosphatase activity, (c) having an embryoid body forming ability, (d) expressing SSEA (Stage-Specific Embryonic Antigen)-1 and SSEA-4, (e) having the same number of chromosomes as does a normal rat cell, (f) capable of being subcultured and holding the undifferentiated state, (g) having in vitro pluripotency, (h) having a potential to differentiate for cells of three embryonic germ lineages, (i) having teratoma formation ability, and (j) having an ability to produce a chimeric rat, a method of establishing the aforementioned rat embryonic stem cell and the like.
Type:
Grant
Filed:
December 18, 2013
Date of Patent:
July 11, 2017
Assignees:
DS Pharma Biomedical Co., Ltd., National Cancer Center
Abstract: Bioimplants and methods of making the bioimplants are provided. The bioimplants comprise biological tissues having conjugated thereto adjunct molecules. The biological tissues are sterilized with a chemical sterilizing agent, such as a water soluble carbodiimide. The processes of making the bioimplants include a process in which an adjunct molecule is conjugated to a biological tissue during the sterilization process.
Type:
Grant
Filed:
January 6, 2009
Date of Patent:
July 4, 2017
Assignee:
Synovis Orthopedic and Woundcare, Inc.
Inventors:
Chandrasekaran Nataraj, Gregg Ritter, Thomas Sander
Abstract: This invention provides the field of therapeutics. Most specifically present invention provides methods of generating in vitro engineered dendritic cells conditionally expressing interleukin-12 (IL-12) under the control of a gene expression modulation system in the presence of activating ligand and uses for therapeutic purposes in animals including human.
Type:
Grant
Filed:
January 9, 2013
Date of Patent:
June 13, 2017
Assignees:
University Of Pittsburgh—Of The Commonwealth System Of Higher Education, Intrexon Corporation
Inventors:
J. Mark Braughler, Prasanna Kumar, Walter J Storkus, Hideho Okada
Abstract: The present invention is directed to constructs, compositions and methods for modulating platelet-type 12 lipoxygenase (12-LO) in adipose tissue in vivo. Specifically, the invention provides constructs encoding expression-inhibiting oligonucleic acids, e.g. antisense and RNA interfering (RNAi) molecules, targeted to a platelet-type 12-LO gene or a transcript thereof, which are capable of reducing or silencing platelet-type 12-LO expression specifically in adipocytes and pre-adipocytes. Vectors comprising these constructs (including, but not limited to, viral vectors), compositions comprising them and methods of using same for the treatment and amelioration of conditions associated with excess fat cell mass and obesity are also provided.
Type:
Grant
Filed:
February 16, 2015
Date of Patent:
May 30, 2017
Assignee:
The Medical Research, Infrastructure, and Health Services Fund of the Tel Aviv Medical Center
Inventors:
Gary Weisinger, Rona Limor, Naftali Stern
Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.
Abstract: Synthetic bacterial messenger RNA can be used to prepare autologous, allogenic or direct nucleic acid cancer vaccines. Cancer cells are transfected either in vitro or in vivo with mRNA obtained from DNA that encodes an immunogenic bacterial protein. An immune response to the cancer is generated from direct administration of the mRNA in vivo or administration of vaccines prepared from cancer cells in vitro.
Type:
Grant
Filed:
May 19, 2016
Date of Patent:
May 2, 2017
Assignee:
MORPHOGENESIS, INC.
Inventors:
Michael J. P. Lawman, Patricia D. Lawman, Meghan Gentilini, Vijay Ramiya, Marina Victor Abdelmaseeh Bastawrous
Abstract: Eukaryotic protein-coding messenger RNAs and non-coding microRNAs are naturally transcribed by type II RNA polymerases (pol-2) but not prokaryotic RNA polymerases. As a result, current eukaryotic RNA and protein production is performed either using eukaryotic pol-2 promoters in hybridomas or mammalian cells or using prokaryotic promoters in bacterial cells. However, because prokaryotic RNA transcription tends to be error-prone, frequent mutation is a big problem. Also, growing hybridomas or mammalian cells is relatively laborious and costly. To overcome these problems, the present invention provides a novel inducible composition and method for producing eukaryotic RNAs and/or their related peptides/proteins directly using eukaryotic pol-2 promoter-driven gene expression in fast growing bacteria, without the need of changing to prokaryotic promoters or growing hybridomas/mammalian cells.
Abstract: A method for production of anti-tumor TRAIL includes inserting a TRAIL molecule, encoded by a viral vector irreversibly derived from a cell line, into a carrier cell, thereby obtaining a stably TRAIL-producing carrier cell, and wherein the TRAIL molecule includes a soluble molecule.
Abstract: The present disclosure provides method of generating cardiomyocytes from post-natal fibroblasts. The present disclosure further provides cells and compositions for use in generating cardiomyocytes.
Abstract: The present disclosure provides method of generating cardiomyocytes from post-natal fibroblasts. The present disclosure further provides cells and compositions for use in generating cardiomyocytes.