Abstract: An expression vector containing appropriate mitochondrion-targeting sequences (MTS) and appropriate 3?UTR sequences provides efficient and stable delivery of a mRNA encoding a protein (CDS) to the mitochondrion of a mammalian cell. The MTS and 3?UTR sequences guide the CDS mRNA from the nuclear compartment of the cell to mitochondrion-bound polysomes, where the CDS is translated. This provides an efficient translocation of a mature functional protein into the mitochondria. A method of targeting mRNA expressed in the nuclear compartment of a mammalian cell to the mitochondrion is also provided. The vector and methods can be used to treat defects in mitochondrial function.
Type:
Grant
Filed:
May 26, 2021
Date of Patent:
July 18, 2023
Assignee:
INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE (INSERM)
Abstract: Provided herein are methods that include introducing into an inner ear of a mammal a therapeutically effective amount of an adeno-associated virus (AAV) vector that includes a nucleotide sequence encoding (a) a polypeptide including an antibody heavy chain variable domain operably linked to a signal peptide and a polypeptide including an antibody light chain variable domain operably linked to a signal peptide; (b) a polypeptide including an antigen-binding antibody fragment operably linked to a signal peptide; or (c) a soluble vascular endothelial growth factor receptor operably linked to a signal peptide.
Type:
Grant
Filed:
August 3, 2021
Date of Patent:
July 11, 2023
Assignee:
Akouos, Inc.
Inventors:
Emmanuel John Simons, Robert Ng, Michael McKenna
Abstract: The present disclosure relates to improved supplements, culture media and methods for enhancing the survival or proliferation of mammalian stem cells. In particular, adding a lipid supplement, such as a lipid-enriched carrier (e.g. a lipid-enriched albumin), to the culture medium may enhance the survival and/or proliferation of the stem cells by at least 5% to 65% as compared to a culture medium that does not contain the lipid supplement.
Type:
Grant
Filed:
January 23, 2018
Date of Patent:
July 11, 2023
Assignee:
STEMCELL TECHNOLOGIES CANADA INC.
Inventors:
Adam Hirst, Arwen Hunter, Melanie Kardel, Wing Chang
Abstract: The present disclosure relates to expansion and activation of T cells. In an aspect, the present disclosure relates to expansion and activation of ?? T cells that may be used for transgene expression. In another aspect, the disclosure relates to expansion and activation of ?? T cells while depleting ?- and/or ?-TCR positive cells. T cell populations comprising expanded ?? T cell and depleted or reduced ?- and/or ?-TCR positive cells are also provided for by the instant disclosure. The disclosure further provides for methods of using the disclosed T cell populations.
Type:
Grant
Filed:
November 26, 2018
Date of Patent:
July 4, 2023
Assignee:
IMMATICS US, INC.
Inventors:
Monique Dao, Steffen Walter, Melinda Mata, Aleksandra Nowicka, Yannick Bulliard, Sarah Missell, Sabrina Kuttruff-Coqui, Norbert Hilf
Abstract: The invention features pancreatic islet and pancreatic organoids, and cell cultures and methods that are useful for the rapid and reliable generation of pancreatic islet and pancreatic islet organoids. The invention also features methods of treating pancreatic diseases and methods of identifying agents that are useful for treatment of pancreatic diseases, such as type 2 diabetes and pancreatic cancer, using the pancreatic islet and pancreatic organoids of the invention.
Type:
Grant
Filed:
March 28, 2022
Date of Patent:
June 27, 2023
Assignee:
Salk Institute for Biological Studies
Inventors:
Ronald Evans, Michael Downes, Annette Atkins, Eiji Yoshihara, Ruth Yu
Abstract: This disclosure relates to compositions comprising human preprimitive streak cells and/or human mesendoderm cells as well as methods for their production. Additionally, disclosed herein are methods of identifying factors useful in the further differentiation of preprimitive streak and mesendoderm cell types.
Type:
Grant
Filed:
December 17, 2019
Date of Patent:
June 6, 2023
Assignee:
ViaCyte, Inc.
Inventors:
Kevin Allen D'Amour, Alan D. Agulnick, Susan Eliazer, Evert Kroon, Emmanuel E. Baetge
Abstract: The current invention relates to an improved method for measuring modulating effects of compounds on epithelial cell barrier function. The methods allows for high throughput screening of test compounds individually or in combination. Compounds improving the epithelial barrier function or compounds negatively influencing epithelial barrier function can be analyzed with the method described herein.
Type:
Grant
Filed:
July 8, 2016
Date of Patent:
May 30, 2023
Assignee:
MIMETAS B.V.
Inventors:
Paul Vulto, Sebastiaan Johannes Trietsch, Henriëtte Leonore Lanz, Marianne Katharina Vormann
Abstract: The invention relates to placenta-derived matrix, methods of preparing, and methods of use thereof. The invention also relates to methods of culturing cells, delivering cells, promoting differentiation of stem cells or tissue-specific progenitor cells, and repairing, replacing, regenerating, filling, reducing or inhibiting scarring of defects using the same. The invention further relates to methods of coating placenta-derived matrix on a surface or injecting the placenta-derived matrix into a site of interest.
Type:
Grant
Filed:
May 27, 2016
Date of Patent:
May 23, 2023
Assignee:
LifeNet Health
Inventors:
Michael Francis, Silvia Chen, Erick Breathwaite, Rudy Rodriguez, Alan Smith, Alexander Huber, Jung Bok Lee
Abstract: The present disclosure provides methods for treating or preventing Huntington's disease (HD) in a subject in need thereof, comprising administering a therapeutic agent that activates mTORC 1 function and/or increases Ras Homolog Enriched in Striatum (Rhes) level in the subject's brain as compared to the function or level in the subject prior to treatment, and methods for modulating mHTT-associated metabolic phenotypes and/or reversal of striatal atrophy by administering a therapeutic agent that activates mTORC1 function and/or increases Ras Homolog Enriched in Striatum (Rhes) level in the subject's brain as compared to the function or level in the subject prior to treatment.
Abstract: The present invention provides a nucleic acid construct comprising the following structure: A-X-B in which A is nucleic acid sequence encoding a first polypeptide which comprises a first signal peptide; B is nucleic acid sequence encoding a second polypeptide which comprises a second signal peptide and X is a nucleic acid sequence which encodes a cleavage site, wherein the first signal peptide or the second signal peptide comprises one or more mutation(s) such that it has fewer hydrophobic amino acids.
Abstract: An object of the present invention is to establish a cell line that is useful as a host cell for use in recombinant protein production, highly expresses transgenes stably, and grows stably. The present invention provides a method for establishing a cell line for recombinant protein production capable of stably expressing two or more foreign genes, comprising transferring a gene of a non-coding RNA suppressing the expression of NfkBia to a cell.
Abstract: Compositions and methods concern the sequence modification of an endogenous genomic DNA region. Certain aspects relate to a method for site-specific sequence modification of a target genomic DNA region in cells comprising: contacting the cells with an activating composition; transfecting the cells with a transfection composition comprising (a) donor DNA and (b) a DNA digesting agent; wherein the donor DNA comprises: (i) a homologous region comprising nucleic acid sequence homologous to the target genomic DNA region; and (ii) a sequence modification region; and wherein the genomic DNA sequence is modified specifically at the target genomic DNA region.
Abstract: The invention discloses methods for the generation of chimaeric human—non-human antibodies and chimaeric antibody chains, antibodies and antibody chains so produced, and derivatives thereof including fully humanised antibodies; compositions comprising said antibodies, antibody chains and derivatives, as well as cells, non-human mammals and vectors, suitable for use in said methods.
Type:
Grant
Filed:
August 9, 2016
Date of Patent:
March 21, 2023
Assignee:
Kymab Limited
Inventors:
Allan Bradley, E-Chiang Lee, Qi Liang, Wei Wang, Glenn Friedrich
Abstract: The present invention relates to pseudotyped retrovirus-like particles or retroviral vectors comprising both engineered envelope glycoproteins derived from a virus of the Paramyxoviridae family fused to a cell targeting domain and fused to a functional domain. The present invention also relates to the use of said pseudotyped retrovirus-like particles or retroviral vectors to selectively modulate the activity of specific subsets of cells, in particular of specific immune cells. These pseudotyped retrovirus-like particles or retroviral vectors are particularly useful for gene therapy, immune therapy and/or vaccination.
Type:
Grant
Filed:
April 20, 2017
Date of Patent:
March 21, 2023
Assignees:
Ecole Normale Superieure de Lyon, Centre National de la Recherche Scientifique (CNRS), Université Claude Bernard Lyon 1, Institut National de la Sante et de la Recherche Medicale
(INSERM)
Inventors:
Caroline Costa Fejoz, Els Verhoeyen, François-Loïc Cosset, Ruben Bender, Christian Buchholz, Qi Zhou
Abstract: The present invention relates to methods and materials useful for systemically delivering polynucleotides across the blood brain barrier using adeno-associated virus as a vector. For example, the present invention relates to methods and materials useful for systemically delivering ?-N-acetylglucosamidinase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of these methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIB. As another example, the present invention relates to methods and materials useful for systemically delivering N-sulphoglucosamine sulfphohydrolase polynucleotides to the central and peripheral nervous systems, as well as the somatic system. Use of this second type of methods and materials is indicated, for example, for treatment of the lysosomal storage disorder mucopolysaccharidosis IIIA.
Abstract: Disclosed are methods for making a vascularized hollow organ derived from human intestinal organoid (HIOs). The HIOs may be obtained from human embryonic stem cells (ESC's) and/or induced pluripotent stem cells (iPSCs), such that the HIO forms mature intestinal tissue. Also disclosed are methods for making a human intestinal tissue containing a functional enteric nervous system (ENS).
Type:
Grant
Filed:
October 16, 2015
Date of Patent:
February 21, 2023
Assignee:
Children's Hospital Medical Center
Inventors:
James M. Wells, Carey Lane Watson, Jorge Orlando Munera, Maxime Mickael Mahe, Michael A. Helmrath, Michael J. Workman
Abstract: The invention discloses methods for the generation of chimaeric human-non-human antibodies and chimaeric antibody chains, antibodies and antibody chains so produced, and derivatives thereof including fully humanised antibodies; compositions comprising said antibodies, antibody chains and derivatives, as well as cells, non-human mammals and vectors, suitable for use in said methods.
Type:
Grant
Filed:
August 4, 2015
Date of Patent:
January 31, 2023
Assignee:
Kymab Limited
Inventors:
Allan Bradley, E-Chiang Lee, Qi Liang, Wei Wang, Glenn Friedrich
Abstract: The present invention relates generally to gene therapy for treating ailments that can affect vision such as retinal degeneration, retinal dystrophy, macular degeneration, macular dystrophy, ischemic retinopathies, and glaucoma. Embodiments include systems and treatments that use AAV-mediated gene therapy or non AAV-mediated DNA, mRNA, or protein therapy to target all retinal cells. An AAV virion can be introduced (e.g., via intravitreal or subretinal injection) into an eye of an individual, or systemically, to express a heterologous gene product such as BMI1 protein (B lymphoma Mo-MLV insertion region 1 homolog).