Abstract: The present invention is directed to methods and compositions comprising novel CRISPR polypeptides and polynucleotides for site-specific cleavage and nicking of nucleic acids, transcriptional control and genome editing.
Type:
Grant
Filed:
August 27, 2015
Date of Patent:
October 22, 2019
Assignee:
NORTH CAROLINA STATE UNIVERSITY
Inventors:
Rodolphe Barrangou, Alexandra E. Briner
Abstract: A trifunctional molecule comprising a target-specific ligand, a ligand that binds a protein associated with the TCR complex and a T cell receptor signaling domain polypeptide is provided. Engineering T cells with this novel receptor engenders antigen specific activation of numerous T cell functions, including cytokine production, degranulation and cytolysis.
Type:
Grant
Filed:
February 6, 2015
Date of Patent:
October 8, 2019
Assignee:
MCMASTER UNIVERSITY
Inventors:
Jonathan Bramson, Christopher W. Helsen, Galina Denisova, Rajanish Giri, Kenneth Anthony Mwawasi
Abstract: The present invention provides fusion proteins including a hyaluronic acid-binding domain of a cartilage matrix protein and a conserved region of a growth factor protein. Certain embodiments provide nucleic nucleic acid sequences encoding a fusion protein and compositions thereof. Methods for using fusion polypeptides and nucleic acid molecules discloses herein are also provided. In certain embodiments, the fusion proteins and/or nucleic acid molecules can be used to treat a cartilage matrix protein-related condition in a subject.
Type:
Grant
Filed:
June 3, 2014
Date of Patent:
October 8, 2019
Assignees:
THE TRUSTEES OF INDIANA UNIVERSITY, UNITED STATES GOVERNMENT AS REPRESENTED BY THE DEPARTMENT OF VETERANS AFFAIRS
Abstract: A nucleic acid containing a dopamine receptor type 2-specific promoter (D2SP) is provided. In certain embodiments, the nucleic acid includes a dopamine receptor type 2-specific promoter (D2SP), wherein the D2SP does not include exon 1 of a D2 receptor gene, wherein the D2SP comprises a Kozak sequence, and wherein the D2SP includes a nucleotide sequence having at least 95% sequence identity to the nucleotide sequence set forth in SEQ ID NO: 1. Also provided are expression vectors, genetically modified host cells and kits that include the subject nucleic acid.
Type:
Grant
Filed:
December 3, 2015
Date of Patent:
October 8, 2019
Assignee:
The Board of Trustees of the Leland Stanford Junior University
Inventors:
Karl A. Deisseroth, Charu Ramakrishnan, Kelly Zalocusky
Abstract: Improved methods of culturing embryos in media having amounts of lactate that have not previously been recognized as beneficial for embryo development. Also, compositions, devices and kits related to the same.
Abstract: Provided herein are nucleic acid amphiphiles and nanostructures such as nanotubes twisted nanotapes and helical nanotapes that comprise the amphiphiles as well as methods to deliver therapeutic agents with the nanostructures.
Type:
Grant
Filed:
June 22, 2015
Date of Patent:
September 17, 2019
Assignee:
Regents of the University of Minnesota
Inventors:
Efrosini Kokkoli, Timothy R. Pearce, Huihui Kuang
Abstract: The present invention relates to an immortalized cell, an immortalized cell line comprising said immortalized cell, a cell culture comprising the immortalized cell or cell line, and a method for the production of an immortalized cell.
Type:
Grant
Filed:
August 12, 2016
Date of Patent:
September 3, 2019
Assignee:
EBERHARD KARLS UNIVERSITAET TUEBINGEN MEDIZINISCHE FAKULTAET
Abstract: A self-regulating gene expression construct comprises a single promoter in operative association with a repressor sequence (e.g., bacterial repressor lacI or gaiR), operator sequence(s) responsive to the expressed repressor protein, and a transgene. A dual-regulating construct comprises a single promoter controlling expression of a bacterial repressor sequence and a transgene, and which, in the presence of a first inducer molecule, transcribes the transgene and repressor; and a ribozyme in association with an aptamer sequence, the aptamer sequence capable of interacting with a second inducer molecule to terminate mRNA degradation by the ribozyme. Also provided are recombinant vectors or viruses containing the self-regulating or dual self-regulating constructs and cells containing the vectors. Such compositions are useful in methods of treating a diseases using gene therapy.
Type:
Grant
Filed:
July 31, 2015
Date of Patent:
August 27, 2019
Assignee:
The Trustees of the University of Pennsylvania
Inventors:
Mitchell Lewis, Jean Bennett, Luk Vandenberghe, Matthew Sochor, Theodore G. Drivas
Abstract: The present invention concerns methods and compositions for immunotherapy employing a modified T cell comprising disrupted T cell receptor and/or HLA and comprising a chimeric antigen receptor. In certain embodiments, the compositions are employed allogeneically as universal reagents for “off-the-shelf treatment of medical conditions such as cancer, autoimmunity, and infection. In particular embodiments, the T cell receptor-negative and/or HLA-negative T cells are generated using zinc finger nucleases, for example.
Type:
Grant
Filed:
November 16, 2012
Date of Patent:
August 27, 2019
Assignee:
BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM
Inventors:
Laurence J. Neil Cooper, Hiroki Torikai
Abstract: The present disclosure provides compositions and methods that rapidly and selectively modify cells of the immune system to achieve therapeutic objectives. The methods can be practiced in vivo and any cell type that expresses a known marker can be targeted for a therapeutic objective.
Abstract: A method of generating a population of cells useful for treating a brain disorder in a subject is disclosed. The method comprises contacting mesenchymal stem cells (MSCs) with at least one exogenous miRNA having a nucleic acid sequence at least 90% identical to a sequence selected from the group consisting of SEQ ID NOs: 15-19 and 27-35, thereby generating a population of cells and/or generating neurotrophic factors that may provide important signals to damaged tissues or locally residing stem cells. MSCs differentiated by miRs may also secrete miRs and deliver them to adjacent cells and therefore provide important signals to neighboring endogenous normal or malignant cells.
Abstract: Methods of producing non-human animal models of corneal angiogenesis and corneal ectatic diseases, such as corneal keratoconus, by applying an aromatic compound to the eye of a non-human animal are described. Also described are non-human animal models of corneal angiogenesis and corneal ectatic diseases, and methods of using the non-human animal models to screen compounds that modulate corneal angiogenesis and corneal ectatic diseases.
Abstract: The present disclosure provides a pharmaceutical composition for treating cancer comprising an RNA oligonucleotide having a particular sequence and structure. Specifically, when a cell line is treated with an RNA oligonucleotide having specific sequence and helical bend structure according to the present disclosure, the expression of ISG56 is increased and apoptosis of cancer cells is induced. Thus, a composition comprising the RNA oligonucleotide can be used as an anticancer agent.
Type:
Grant
Filed:
October 14, 2016
Date of Patent:
July 2, 2019
Assignee:
KOREA ADVANCED INSTITUTE OF SCIENCE AND TECHNOLOGY
Abstract: This invention relates, e.g., to a molecular delivery system comprising A. a substrate having a nanostructured surface region which comprises a plurality of nanostructures and, covalently attached to the substrate, multiple copies of a first member of a binding pair; and B. at least one vector nanoparticle which comprises, encapsulated therein, a molecule of interest, and on its surface, multiple copies of second member of the binding pair. Methods of using the molecular delivery system to deliver a molecule of interest to a cell are also described.
Type:
Grant
Filed:
July 23, 2012
Date of Patent:
July 2, 2019
Assignees:
The Regents of the University of California, National Institutes of Health (NIH)
Abstract: This disclosure relates to recombinant cellular expression of chimeric proteins with peptide sequences derived from lymphocyte receptors and uses for treating cancer. In certain embodiments, the disclosure relates to a recombinant vector comprising a nucleic acid that encodes a chimeric protein with a segment with a targeting moiety based on a variable lymphocyte receptor (VLR) capable of binding a tumor associated antigen and a segment with a T cell signal transduction subunit. In certain embodiments, the recombinant vectors are used in immune based cancer treatments.
Type:
Grant
Filed:
February 9, 2015
Date of Patent:
June 18, 2019
Assignees:
Emory University, Children's Healthcare of Atlanta
Inventors:
H. Trent Spencer, Christopher B. Doering, Brantley R. Herrin, Max Dale Cooper
Abstract: Disclosed herein are microvessel endothelial cell surface markers and methods, compositions, agents, and kits relating to those surface markers.
Type:
Grant
Filed:
July 9, 2014
Date of Patent:
June 4, 2019
Assignees:
President and Fellows of Harvard College, The Brigham and Women's Hospital
Inventors:
Ulrich H. von Andrian, Aude Thiriot, Omid Farokhzad, Jinjun Shi
Abstract: Disclosed are nanoparticles that are introduced into cells and express a specific protein and a manufacturing method thereof. More particularly, the present invention relates to mRNA nanoparticles, which increase the expression of a specific protein capable of stimulating the cellular immune system to induce cellular immune responses and are thus applicable to treat a variety of diseases, do not require passage across the nuclear envelope because a desired gene is delivered not as plasmid DNA itself but in the form of mRNA, thus improving the efficiency of protein expression, and the nanoparticles are generated through a one-step process with a relatively small amount of plasmid DNA via rolling circle transcription (RCT), thereby providing a simple and economical process for gene delivery. The present invention is also concerned with such mRNA nanoparticles.
Type:
Grant
Filed:
October 1, 2015
Date of Patent:
May 28, 2019
Assignee:
UNIVERSITY OF SEOUL INDUSTRY COOPERATION
Abstract: This disclosure describes a composition and method of magnetic nanoparticles (MNP) that are bound to a baculovirus (BV). The MNP-BV can be systemically administered to a patient, and a strong magnetic field applied to the target tissue, thus allowing uptake and expression only in the target tissue. Off-target effects are not seen because the MNP-BC is inactivated by the complement system outside of the magnetic field.
Abstract: The present invention relates to methods of treating diabetes in a human subject comprising the use of pancreatic islets or of embryonic pancreatic tissue of a transgenic animal, wherein said transgenic animal contains a polynucleotide sequence encoding a CTLA4 peptide-immunoglobulin fusion, preferably LEA29Y, and expresses said CTLA4 peptide-immunoglobulin fusion in a tissue-specific manner in pancreatic islets.
Type:
Grant
Filed:
October 27, 2016
Date of Patent:
May 14, 2019
Assignee:
MWM BIOMODELS GMBH
Inventors:
Eckhard Wolf, Nikolai Klymiuk, Lelia Wolf-Van Buerck, Jochen Seißler
Abstract: Provided herein are recombinant constructs, vectors and expression cassettes including a first promoter which is suitably a tRNA promoter operably connected to a first polynucleotide encoding a first single guide RNA and a second promoter operably connected to a second polynucleotide encoding a Cas9 polypeptide. The first single guide RNA includes a first portion complementary to a strand of a target sequence of a DNA virus and a second portion capable of interacting with the Cas9 polypeptide. Also provided are codon optimized Staphylococcus aureus derived Cas9 polynucleotides and polypeptides with nuclear localization signals and optionally an epitope tag. Also provided are constructs for production of sgRNAs including a tRNA. Methods of inhibiting viral replication, inhibiting expression of a target sequence from a virus or treating a viral infection or viral induced cancer using the compositions are also provided.
Type:
Grant
Filed:
February 18, 2015
Date of Patent:
May 14, 2019
Assignees:
Duke University, Emory University, The United States of America as Represented by the Department of Veterans Affairs
Inventors:
Bryan R. Cullen, E. Matthew Kennedy, Hal P. Bogerd, Anand Kornepati, Adam Mefferd, Raymond F. Schinazi