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 method and vectors for controlling blood glucose levels in a mammal are disclosed. In one embodiment, the method comprises the steps of: treating the hepatocyte cells of a patient with a first, second or third vector, wherein the first vector comprises a promoter enhancer, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase and an albumin 3?UTR and lacks an HGH intron, wherein the second vector comprises an HGH intron, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase site and an albumin 3?UTR and lacks a promoter enhancer, wherein the third vector comprises an HGH intron, glucose inducible regulatory elements, a liver-specific promoter, a gene encoding human insulin with modified peptidase site, an albumin 3?UTR and a promoter enhancer and observing the patient's insulin levels, wherein the patient's insulin levels are controlled.
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: The present invention provides for nucleic acids improved for the expression of interleukin-15 (IL-15) in mammalian cells. The invention further provides for methods of expressing IL-15 in mammalian cells by transfecting the cell with a nucleic acid sequence encoding an improved IL-15 sequence. The present invention further provides expression vectors, and IL-15 and IL-15 receptor alpha combinations (nucleic acid and protein) that increase IL-15 stability and potency in vitro and in vivo. The present methods are useful for the increased bioavailability and biological effects of IL-15 after DNA, RNA or protein administration in a subject (e.g. a mammal, a human).
Type:
Grant
Filed:
October 9, 2017
Date of Patent:
October 1, 2019
Assignee:
THE UNITED STATES OF AMERICA, AS REPRESENTED BY THE SECRETARY, DEPARTMENT OF HEALTH AND HUMAN SERVICES
Abstract: The present invention is directed to a composition and method which to treat diseases and to enhance a regulated immune response. More particularly, the present invention is drawn to compositions that are based on dendritic cells modified to express an inducible form of a co-stimulatory polypeptide.
Type:
Grant
Filed:
January 5, 2017
Date of Patent:
September 24, 2019
Assignee:
Baylor College of Medicine
Inventors:
David Spencer, Brent Hanks, Kevin Slawin
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: The invention provides methods of inducing or improving responsiveness to a VEGF antagonist to a subject or a subject population comprising administering an adenovirus comprising a nucleic acid construct comprising a F AS-chimera gene operably linked to an endothelial cell-specific promoter and administering the VEGF antagonist.
Type:
Grant
Filed:
May 26, 2017
Date of Patent:
August 20, 2019
Assignee:
Vascular Biogenics Ltd.
Inventors:
Andrea Rachel Leubitz, Naamit Sher, Erez Feige, Eyal Breitbart
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 invention relates to at least one agent capable of increasing the level of one or more miRNA in a cell or cells of a subject, said miRNA comprising the sequence UUCCCUU, for use in the treatment and/or prevention of a retinal dystrophy, in particular characterized by photoreceptor degeneration, relative pharmaceutical compositions, nucleic acids, vectors and host cells.
Type:
Grant
Filed:
March 11, 2014
Date of Patent:
August 6, 2019
Assignee:
FONDAZIONE TELETHON
Inventors:
Sandro Banfi, Enrico Maria Surace, Ivan Conte, Marianthi Karali, Elena Marrocco
Abstract: The invention relates to means and methods for removing a proteolytic cleavage site from a protein comprising providing a cell that expresses pre-mRNA encoding the protein with an anti-sense oligonucleotide that induces skipping of the exonic sequence that encodes the proteolytic cleavage site, the method further comprising allowing translation of mRNA produced from the pre-mRNA.
Abstract: The invention relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides encoding AADC for the treatment of Parkinson's Disease.
Type:
Grant
Filed:
November 5, 2015
Date of Patent:
July 2, 2019
Assignee:
VOYAGER THERAPEUTICS, INC.
Inventors:
Robert Kotin, Adrian Philip Kells, Bernard Ravina
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: 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: In certain aspects the invention provides immunogenic compositions comprising CH848 HIV-1 envelopes and their use in methods to induce immune responses in subjects, e.g., human subjects.
Type:
Grant
Filed:
March 31, 2015
Date of Patent:
June 18, 2019
Assignees:
DUKE UNIVERSITY, TRIAD NATIONAL SECURITY, LLC, THE TRUSTEES OF THE UNIVERSITY OF PENNSYLVANIA
Inventors:
Barton F. Haynes, Beatrice H. Hahn, George M. Shaw, Bette T. Korber, Peter T. Hraber
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: The present disclosure relates to an efficient genome editing technique. In one aspect, the technique can greatly improve the efficiency of homologous recombination during intracellular targeting, including gene targeting. Using this technique, genetically modified cell lines, rat, mouse, zebrafish, and fertilized eggs of other species can be quickly and efficiently generated.
Abstract: The present disclosure provides a synthetic nanoparticle comprising a peptide nucleic acid (PNA) oligomer conjugated to a lipid, wherein the PNA oligomer noncovalently complexes with an immunomodulatory compound, thereby forming a nanoparticle. The nanoparticles are useful to elicit immune responses and can be used to treat a broad range of cancers and infectious diseases.
Abstract: Disclosed are an anionic drug-containing polymeric micelle particle comprising: an anionic drug as an active ingredient; a cationic lipid; and an amphiphilic block copolymer, wherein the anionic drug forms a complex with the cationic lipid, and the complex is entrapped in the micelle structure of the amphiphilic block copolymer, and a method for preparing the same. The particle may increase stability of the anionic drug in blood or in a body fluid, and it may enable intracellular delivery to improve efficacy of anionic drugs.
Type:
Grant
Filed:
April 25, 2016
Date of Patent:
May 21, 2019
Assignee:
SAMYANG BIOPHARMACEUTICALS CORPORATION
Inventors:
Se-Ho Kim, Ji-Yeon Son, Muhn-Ho La, Sung-Won Choi, Min-Hyo Seo