Abstract: The present disclosure provides gene edited modified immune cells or precursors thereof (e.g., gene edited modified T cells) comprising an exogenous T cell receptor (TCR) and/or a chimeric antigen receptor (CAR) having specificity for a target antigen, and an insertion and/or deletion in one or more endogenous gene loci, wherein the endogenous gene loci encode regulators of T cell function, thereby resulting in immune cells having enhanced function. Compositions and methods of treatment are also provided. The present invention provides methods of screening for TCR- or CAR-T cells with enhanced immune function (e.g., T cell efficacy, T cell memory, and/or T cell persistence).
Type:
Grant
Filed:
January 29, 2024
Date of Patent:
May 6, 2025
Assignee:
The Trustees of the University of Pennsylvania
Abstract: Provided herein are methods and compositions comprising a bacterium or a metabolite thereof for enhancing mitochondrial and/or peroxisomal function.
Type:
Grant
Filed:
July 12, 2023
Date of Patent:
April 29, 2025
Assignee:
The General Hospital Corporation
Inventors:
Gary Ruvkun, J. Amaranath Govindan, Elamparithi Jayamani
Abstract: The present invention discloses a recombinant fused polypeptide, a preparation method therefor, and use thereof. The recombinant fused polypeptide is represented by the following general formula: X-linker1-Y; Y-linker1-X; X-linker2-Y; Y-linker2-X, where X is PRCWRGEGGGGIVRRADRAAVPGGGGRGD; and Y is Acetyl-SDKPGGGGTSLDASIIWAMMQNGGGGLSKL. The recombinant fused polypeptide according to the present invention can treat various fibrosis diseases and symptoms, and therapeutic use includes anti-pulmonary fibrosis, anti-hepatic fibrosis, anti-skin fibrosis, anti-renal fibrosis, anti-myocardial fibrosis, and resistance to lung tissue lesions.
Abstract: A tandem DNA element capable of enhancing protein synthesis efficiency, in particular, the nucleic acid construct is formed by an IRES enhancer (such as ScBOI1, ScFLO8, ScNCE102, ScMSN1, KlFLO8, KlNCE102, KlMSN1, KlBOI1) derived from eukaryotic cells (such as yeast), a ? sequence, and a yeast-specific Kozak sequence in tandem. The use of the nucleic acid construct in a yeast-based in vitro biosynthesis system (such as a yeast-based in vitro protein synthesis system) can significantly improve protein synthesis efficiency.
Abstract: Disclosed is a method for producing, in one step, “made to measure” double-stranded DNA vectors from molecular bricks including sequences of interest in the presence of a one and only type IIs restriction enzyme.
Type:
Grant
Filed:
February 10, 2016
Date of Patent:
April 15, 2025
Assignees:
Universite de Lille, Centre National de la Recherche Scientifique (CNRS)
Inventors:
Geoffroy De Bettignies, Carine De Bettignies, Sylvain Julien
Abstract: Engineered nucleic acids encoding genome editing system components are provided, as are engineered RNA-guided nucleases that include inserts encoded in part by cellular genomic or other sequences recognized by guide RNAs.
Type:
Grant
Filed:
May 12, 2023
Date of Patent:
April 1, 2025
Assignee:
EDITAS MEDICINE, INC.
Inventors:
Ari E. Friedland, Hariharan Jayaram, Barrett Ethan Steinberg
Abstract: A mammalian or avian cell line that expresses high levels of human influenza virus receptors is provided. In one embodiment, the cell line supports human influenza virus, e.g., human A/H3 influenza virus, isolation and growth much more effectively than corresponding conventional (unmodified) cells or in corresponding human virus receptor-overexpressing cells, and the propagated viruses may maintain higher genetic stability than in the corresponding cells.
Type:
Grant
Filed:
November 30, 2023
Date of Patent:
March 25, 2025
Assignees:
Wisconsin Alumni Research Foundation (WARF), The University of Tokyo
Abstract: The invention relates to a new method of characterising a target polynucleotide. The method uses a pore and a Dda helicase. The helicase controls the movement of the target polynucleotide through the pore. The invention also relates to modified Dda helicases which can be used to control the movement of polynucleotides and are particularly useful for sequencing polynucleotides.
Type:
Grant
Filed:
October 17, 2022
Date of Patent:
March 25, 2025
Assignee:
Oxford Nanopore Technologies PLC
Inventors:
Mark Bruce, Andrew John Heron, Ruth Moysey, Szabolcs Soeroes, Elizabeth Jayne Wallace, James White
Abstract: The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems.
Type:
Grant
Filed:
December 27, 2022
Date of Patent:
March 25, 2025
Assignees:
THE BROAD INSTITUTE, INC., MASSACHUSETTS INSTIT JTE OF TECHNOLOGY, UNIVERSITY OF TOKYO, PRESIDENT AND FELLOWS OF HARVARD COLLEGE
Inventors:
Silvana Konermann, Alexandro Trevino, Mark Brigham, Fei Ran, Patrick Hsu, Chie-Yu Lin, Osamu Nureki, Hiroshi Nishimasu, Ryuichiro Ishitani, Feng Zhang
Abstract: The invention includes a mutant Taq polymerase, which can significantly extend and amplify a target sequence where the extension conditions are time limited to as little as one second. The mutant Taq polymerase, or a biologically active fragment thereof, has one or more substitutions differing from the wild type as shown in Table I.
Abstract: The invention relates to modified helicases with reduced unbinding from polynucleotides. The helicases can be used to control the movement of polynucleotides and are particularly useful for sequencing polynucleotides.
Type:
Grant
Filed:
October 18, 2022
Date of Patent:
March 18, 2025
Assignee:
Oxford Nanopore Technologies PLC
Inventors:
Andrew John Heron, James Anthony Clarke, Ruth Moysey, Elizabeth Jayne Wallace, Mark John Bruce, Lakmal Jayasinghe, Domenico Caprotti, Szabolcs Soeroes, Luke McNeill, David Antoni Alves, Rebecca Victoria Bowen, John Milton
Abstract: The present invention provides DNA polymerases that are highly resistant to inhibitors, and that can shorten the entire nucleic acid amplification reaction time by shortening the reverse transcription reaction time in a nucleic acid amplification method, in particular, in PCR or RT-PCR. The DNA polymerase is characterized by having reverse transcription activity, and comprising at least one amino acid modification at position 509 or 744 in SEQ ID NO: 1 or 2. In particular, the amino acid modification at position 509 or 744 in SEQ ID NO: 1 or 2 is substitution with histidine, lysine, or arginine.
Abstract: The present disclosure provides for genetically modified organisms that provide numerous health benefits but also have an improved flavor profile and a more palatable aroma for the consumer of the organism.
Abstract: Cas-protein-ready tau biosensor cells, CRISPR/Cas synergistic activation mediator (SAM)-ready tau biosensor cells, and methods of making and using such cells to screen for genetic modifiers of tau seeding or aggregation are provided. Reagents and methods for sensitizing such cells to tau seeding activity or tau aggregation or for causing tau aggregation are also provided.
Type:
Grant
Filed:
November 6, 2023
Date of Patent:
January 28, 2025
Assignee:
Regeneron Pharmaceuticals, Inc.
Inventors:
Marine Prissette, Matthew Koss, Wen Fury, Brian Zambrowicz
Abstract: Disclosed are compositions, systems, kits, and methods for detecting an analyte or target molecule in a sample by regulated in vitro transcription. The compositions, systems, kits, and methods typically comprise and/or utilize one or more components selected from: (a) an RNA polymerase; (b) an allosteric transcription factor (ATT), wherein the ATT binds an analyte or target molecule as a ligand; (c) an engineered transcription template; and/or any combination thereof. The engineered transcription template typically comprises a promoter sequence for the RNA polymerase and an operator sequence for the ATT. The promoter sequence and operator sequence are operably linked to a sequence encoding an RNA, wherein the ATT modulates transcription of the encoded RNA when the ATT binds the analyte or target molecule as a ligand, wherein the transcribed RNA generates a detectable signal in conjunction with a reporter molecule.
Type:
Grant
Filed:
November 11, 2019
Date of Patent:
January 21, 2025
Assignee:
Northwestern University
Inventors:
Julius B. Lucks, Khalid K. Alam, Jaeyoung K. Jung
Abstract: A crystal structure of a Non-LTR-retroelement reverse transcriptase and methods of using the same to identify enzymes with improved activity are provided. Mutant reverse transcriptase enzymes and methods of using the same are also provided.
Type:
Grant
Filed:
May 10, 2022
Date of Patent:
January 21, 2025
Assignee:
BOARD OF REGENTS, THE UNIVERSITY OF TEXAS SYSTEM
Inventors:
Jennifer L. Stamos, Alfred M. Lentzsch, Seung Kuk Park, Georg Mohr, Alan M. Lambowitz
Abstract: Methods and compositions for making bacteriocins are described in some embodiments herein. In some embodiments, pro-polypeptide comprising the bacteriocins in the desired ratios in cis, and separated by cleavage sited can be produced by a microbial cell comprising a nucleic acid encoding the pro-polypeptide. In some embodiments microfluidic devices and methods for making specified mixtures of antimicrobial peptides and/or bacteriocins are described.
Abstract: Provided are compositions and methods for enzyme replacement therapy using modified human cystathionine beta synthase (CBS) in the treatment of homocystinuria and related diseases and disorders.
Type:
Grant
Filed:
August 23, 2023
Date of Patent:
January 14, 2025
Assignee:
The Regents of the University of Colorado, A Body Corporate
Inventors:
Jan P. Kraus, Tomas Majtan, Erez Bublil
Abstract: The invention concerns methods for preventing the reduction of disulfide bonds during the recombinant production of disulfide-containing polypeptides. In particular, the invention concerns the prevention of disulfide bond reduction during harvesting of disulfide-containing polypeptides, including antibodies, from recombinant host cell cultures.
Type:
Grant
Filed:
September 4, 2024
Date of Patent:
December 24, 2024
Assignee:
Genentech, Inc.
Inventors:
Yung-Hsiang Kao, Michael W. Laird, Melody Trexler Schmidt, Rita L. Wong, Daniel P. Hewitt
Abstract: Provided are MTSP-1 polypeptides modified to have altered activity and/or specificity so that they cleave a complement protein, such as complement protein C3, to inhibit its activity and thereby inhibit complement activation. The modified MTSP-1 polypeptides that inhibit complement activation can be used for treatment of diseases and conditions in which complement activation plays a role. Such diseases and conditions include inflammatory diseases and diseases with an inflammatory component. Exemplary of these disorders are ischemic and reperfusion disorders, including myocardial infarction and stroke, sepsis, autoimmune diseases, ophthalmic disorders, such as diabetic retinopathies and macular degeneration, including age-related macular degeneration (AMD), and transplanted organ rejection, such as renal delayed graft function (DGF).
Type:
Grant
Filed:
August 16, 2023
Date of Patent:
December 24, 2024
Assignee:
Vertex Pharmaceuticals, Incorporated
Inventors:
Edwin L. Madison, Vanessa Soros, Mikhail Popkov