Abstract: Methods are described for predicting ancestral sequences for viruses or portions thereof. Also described are predicted ancestral sequences for adeno-associated virus (AAV) capsid polypeptides. The disclosure also provides methods of gene transfer and methods of vaccinating subjects by administering a target antigen operably linked to the AAV capsid polypeptides.
Type:
Grant
Filed:
July 29, 2016
Date of Patent:
August 11, 2020
Assignees:
Massachusetts Eye and Ear Infirmary, Schepens Eye Research Institute
Abstract: The present invention relates to the field of genetic engineering and more particularly to nucleic acid editing and genome modification. The present invention provides an isolated Cas protein or polypeptide fragment thereof having an amino acid sequence of SEQ ID NO: 1 or a sequence of at least 77% identity therewith, wherein the Cas protein or polypeptide is capable of DNA cleavage at a temperature in the range 50° C. and 100° C. inclusive. The invention further provides isolated nucleic acid molecules encoding said Cas9 nucleases, expression vectors and host cells. The Cas9 nucleases disclosed herein provide novel tools for genetic engineering at elevated temperatures and are of particular value in the genetic manipulation of thermophilic organisms; particularly microorganisms.
Type:
Grant
Filed:
June 6, 2016
Date of Patent:
August 4, 2020
Assignee:
Purac Biochem B.V.
Inventors:
John Van Der Oost, Martinus Johannes Arnoldus Daas, Servatius Wilhelmus Maria Kengen, Willem Meindert De Vos
Abstract: This invention relates generally to populations of microvesicles containing or otherwise associated with viral particles, methods of producing these purified populations, and methods of using these purified populations in a variety of diagnostic, therapeutic and/or prophylactic indications.
Abstract: The invention provides a recombinant polypeptide X—Y for enhancing cell transduction efficiency of a target agent, wherein X is a cell penetrating peptide DPV3, and Y is an Hsp40-J domain. Also provided is a method for enhancing cell transduction efficiency of a target agent, comprising conjugating/attaching said target agent with a recombinant polypeptide X—Y, wherein X is a cell penetrating peptide DPV3, and Y is an Hsp40-J domain. Further provided is a pharmaceutical composition comprising a therapeutic agent, wherein said therapeutic agent is modified by conjugating/attaching with a recombinant polypeptide X—Y, wherein X is a cell penetrating peptide DPV3, and Y is an Hsp40-J domain.
Type:
Grant
Filed:
December 15, 2016
Date of Patent:
July 21, 2020
Assignee:
AGRICULTURAL TECHNOLOGY RESEARCH INSTITUTE
Inventors:
Chin-Kai Chuang, Yu-Hsiu Su, Tzuyin Lin
Abstract: Described herein are human transgenic beta cells expressing fugetactic levels of CXCL12 to a subject in need thereof. Also described herein are beta cells comprising a transgene comprising a nucleic acid sequence encoding CXCL12.
Abstract: The present invention relates to the field of genetic engineering and more particularly to nucleic acid editing and genome modification. The present invention provides an isolated Cas protein or polypeptide fragment thereof having an amino acid sequence of SEQ ID NO: 1 or a sequence of at least 77% identity therewith, wherein the Cas protein or polypeptide is capable of DNA cleavage at a temperature in the range 50° C. and 100° C. inclusive. The invention further provides isolated nucleic acid molecules encoding said Cas9 nucleases, expression vectors and host cells. The Cas9 nucleases disclosed herein provide novel tools for genetic engineering at elevated temperatures and are of particular value in the genetic manipulation of thermophilic organisms; particularly microorganisms.
Type:
Grant
Filed:
June 6, 2016
Date of Patent:
June 23, 2020
Assignee:
Purac Biochem B.V.
Inventors:
John Van Der Oost, Martinus Johannes Arnoldus Daas, Servatius Wilhelmus Maria Kengen, Willem Meindert De Vos
Abstract: Disclosed are adeno-associated virus (AAV) vectors comprising a nucleotide sequence encoding RP2 or RPGR-ORF15 and related pharmaceutical compositions. Also disclosed are methods of treating or preventing X-linked retinitis pigmentosa, increasing photoreceptor number in a retina of a mammal, and increasing visual acuity of a mammal using the vectors and pharmaceutical compositions.
Type:
Grant
Filed:
March 11, 2016
Date of Patent:
May 12, 2020
Assignee:
The United States of America, as represented by the Secretary, Department of Health and Human Services
Abstract: The present invention relates to a method for the generation of VH heavy chain-only antibodies in a transgenic non-human mammal. In particular, the present invention relates to a method for the production of a VH heavy chain-only antibody in a transgenic non-human mammal comprising the step of expressing more than one heterologous VH heavy chain locus in that mammal.
Type:
Grant
Filed:
December 8, 2015
Date of Patent:
May 5, 2020
Assignee:
Erasmus University Medical Center
Inventors:
Franklin Gerardus Grosveld, Richard Wilhelm Janssens, Roger Kingdon Craig
Abstract: Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve use of a FXN coding sequence, a truncated FXN 3? UTR, and a promoter.
Type:
Grant
Filed:
April 23, 2016
Date of Patent:
April 14, 2020
Assignee:
University of Florida Research Foundation, Incorporated
Abstract: The present invention provides that exosomes from human body fluid samples contain double stranded genomic DNA that spans all chromosomes and may be used to determine the mutation status of genes of interest in diseases, such as cancer. Furthermore, the present invention provides the use of exosomes to produce therapeutic proteins and for their use in therapy as well as the detection of cancer cell-derived exosomes to diagnose cancer and monitor therapeutic response.
Type:
Grant
Filed:
November 1, 2017
Date of Patent:
March 24, 2020
Assignee:
Board of Regents, The University of Texas System
Abstract: The invention relates to LAH4 peptides and functional derivatives thereof and their use for improving transduction efficiency of viruses into target cells.
Type:
Grant
Filed:
June 28, 2012
Date of Patent:
March 24, 2020
Assignees:
Genethon, Centre National de la Recherche Scientique, Institut National de la Sainte et de la Recherche Madicale
Inventors:
David Fenard, Antoine Kichler, Samia Martin
Abstract: Described are methods for removing a proteolytic cleavage site, the HCHWA-D mutation or the amino acids encoded by a trinucleotide repeat expansion 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 comprises the proteolytic cleavage site, HCHWA-D mutation or trinucleotide repeat expansion, respectively, the method further comprising allowing translation of mRNA produced from the pre-mRNA.
Abstract: The present invention provides compositions and methods for regulating the specificity and activity of immune effector cells for use in immunotherapy. In one embodiment, the invention provides a type of chimeric antigen receptor (CAR) wherein the CAR is termed a “NKR-CAR” which is a CAR design comprising a component of a receptor naturally found on natural killer (NK) cells. In one embodiment, the NK receptor includes but is not limited to a naturally occurring activating and inhibitory receptor of NK cells known as a killer cell immunoglobulin-like receptor (KIR).
Type:
Grant
Filed:
September 17, 2015
Date of Patent:
March 3, 2020
Assignees:
Novartis AG, The Trustees of the University of Pennsylvania
Inventors:
Gregory Beatty, Boris Engels, Neeraja Idamakanti, Carl H. June, Andreas Loew, Michael C. Milone, Huijuan Song, Enxiu Wang, Qilong Wu
Abstract: Provided are nucleic acid sequences, recombinant adeno-associated viral particles, compositions, and methods related to treating cone monochromacies, such as blue cone monochromacy (BCM). Specifically, the nucleic acid sequences, recombinant adeno-associated viral particles, compositions, and methods involve use of an M-opsin gene operably linked to a cone-specific promoter. Further disclosed is the sequence of a cone-specific PR2.1 promoter.
Type:
Grant
Filed:
March 18, 2016
Date of Patent:
January 14, 2020
Assignee:
University of Florida Research Foundation, Incorporated
Abstract: Methods of the invention encompass delivery of nucleic acid sequences encoding ABCD1 for the treatment of X-linked Adrenoleukodystrophy (X-ALD), e.g., for Adrenomyeloneuropathy (AMN).
Abstract: Mutant mammalian RPE65 proteins and portions thereof, and nucleic acids encoding the mutants, for use in treating a condition related to retinal degeneration in a subject, the mutant mammalian RPE65 proteins or portions thereof having isomerohydrolase activity. A gene therapy method of treating a condition related to retinal degeneration in a mammalian subject in need of such treatment, comprising administering to the subject a therapeutically-effective amount of a vector comprising a nucleic acid encoding a mutant mammalian RPE65 protein or a portion thereof. A method of treating a condition related to retinal degeneration in a subject in need of such treatment, comprising administering to the subject a therapeutically-effective amount of at least one of a mutant mammalian RPE65 protein or a portion thereof.
Type:
Grant
Filed:
July 27, 2015
Date of Patent:
December 3, 2019
Assignee:
The Board of Regents of the University of Oklahoma
Abstract: The present application generally relates to methods of genetically modifying a T-cell comprising a chimeric antigen receptor wherein the T-cell lacks a co-receptor for HIV. The application further relates to methods of making a nucleic acid encoding a chimeric antigen receptor, nucleic acids encoding a chimeric antigen receptor, and genetically modified T-cells comprising a chimeric antigen receptor disclosed herein. The application further relates to methods of treating, inhibiting, or ameliorating HIV in a subject including administering to the subject a cell disclosed herein.
Type:
Grant
Filed:
April 8, 2015
Date of Patent:
December 3, 2019
Assignee:
Seattle Children's Hospital
Inventors:
Thor Wagner, Andrew M. Scharenberg, David J. Rawlings, Blythe Sather, Jaya Sahni
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.