Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.
Abstract: Methods and compositions for the rapid and reversible destabilizing of specific proteins using cell-permeable, synthetic molecules are described. Stability-affecting proteins, e.g., derived from FKBP and DHFR proteins are fused to a protein of interest and the presence or absence of the ligand is used to modulate the stability of the fusion protein.
Type:
Grant
Filed:
March 22, 2012
Date of Patent:
November 8, 2016
Assignee:
The Board of Trustees of the Leland Stanford Junior University
Inventors:
Thomas J. Wandless, Laura Anne Banaszynski, Mari Iwamoto, Lystranne Alysia Maynard, Ling-Chun Chen
Abstract: A method of rapidly inducing large-scale and high-purity mesenchymal stem cells to transdetermine into hematopoietic stem cells is provided with the steps of preparing homogeneous medium of mesenchymal stem cells; combining a plurality of small RNA molecules; assembling and transfecting nanoparticles of nucleic acids and polypeptides; inducing and amplifying medium of post-transdetermined hematopoietic stem cells; and activating a plurality of hemopoiesis-related genes.
Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.
Abstract: Stimulation of target cells using light, e.g., in vivo or in vitro, is implemented using a variety of methods and devices. One example involves a vector for delivering a light-activated molecule comprising a nucleic acid sequence that codes for light-activated molecule. The light-activated molecule includes a modification to a location near the all-trans retinal Schiff base, e.g., to extends the duration time of the open state. Other aspects and embodiments are directed to systems, methods, kits, compositions of matter and molecules for ion channels or pumps or for controlling currents in a cell (e.g., in in vivo and in vitro environments).
Type:
Grant
Filed:
February 19, 2014
Date of Patent:
October 4, 2016
Assignee:
The Board of Trustees of the Leland Stanford Junior University
Inventors:
Karl Deisseroth, Ofer Yizhar, Lisa Gunaydin, Peter Hegemann, Andre Berndt
Abstract: The present inventions describes a method that, starting from pluripotent cells, leads to the obtainment, in a reproducible and efficient manner, of endodermal cells precursor. These cells reveal useful also for application in the regenerative therapy.
Abstract: Provided are compositions and methods for delivering biological moieties such as modified nucleic acids into cells to modulate protein expression. Such compositions and methods include the use of modified messenger RNAs, and are useful to treat or prevent diseases, disorders or conditions, or to improve a subject's heath or wellbeing.
Type:
Grant
Filed:
October 8, 2015
Date of Patent:
September 20, 2016
Assignee:
Moderna Therapeutics, Inc.
Inventors:
Jason P. Schrum, Stephane Bancel, Noubar B. Afeyan, Kenechi Ejebe
Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.
Abstract: The present invention provides an intraceptor that interacts with and decreases activity of with VEGF and/or a VEGFR for the treatment of angiogenesis-related conditions. The present invention further provides pharmaceutical compositions, and methods of use thereof, for the treatment and prevention of an angiogenesis-related condition using said intraceptors. The invention further provides for nucleic acids encoding said intraceptors.
Type:
Grant
Filed:
March 31, 2014
Date of Patent:
September 20, 2016
Assignees:
Medical College of Georgia Research Institute, University of Utah Research Foundation
Abstract: A generally spherical growth factor sensitive vesicle bearing growth factor receptors (12) having chemical compounds (14) covalently cross-linked to their non-growth factor binding domains. The chemical compounds are capable of associating to form larger chemical compounds (16) capable of destabilizing the vesicle. Methods for treating growth factor-overexpressing neoplasms are disclosed.
Abstract: The technology relates in part to compositions comprising modified Caspase-9 polypeptides, compositions comprising nucleic acids coding for modified Caspase-9 polypeptides, chimeric modified Caspase-9 polypeptides, and methods of use thereof, including methods for cell therapy. Methods for cell therapy include modifying transfused cells to express an inducible modified Caspase-9 protein, with reduced basal activity in the absence of the inducer.
Abstract: The invention provides compositions and methods of treating subjects afflicted with a photoreceptor disorder. Methods for treating a subject suffering from a disorder characterized by photoreceptor cell degeneration are provided, wherein a gene encoding a photosensitive protein is introduced into a retinal cell of a subject. In one aspect of the invention, the retinal cells which receive the photosensitive protein include non-photoreceptor cells such as horizontal cells, amacrine cells, bipolar cells, and ganglion cells.
Abstract: An optimized coding sequence of human blood clotting factor eight (VIII) and a promoter may be used in vectors, such as rAAV, for introduction of factor VIII, and/or other blood clotting factors and transgenes. Exemplary of these factors and transgenes are alpha-1-antitrypsin, as well as those involved in the coagulation cascade, hepatocye biology, lysosomal storage, urea cycle disorders, and lipid storage diseases. Cells, vectors, proteins, and glycoproteins produced by cells transformed by the vectors and sequence, may be used in treatment.
Type:
Grant
Filed:
July 8, 2010
Date of Patent:
July 19, 2016
Assignees:
UCL BUSINESS PLC, THROMBOSIS RESEARCH INSTITUTE, ST. JUDE CHILDREN'S RESEARCH HOSPITAL
Inventors:
Amit Nathwani, Natalie Ward, Adrian Thrasher, Edward Tuddenham, John McVey, John Gray, Andrew Davidoff
Abstract: The present invention relates in part to nucleic acids encoding proteins, therapeutics comprising nucleic acids encoding proteins, methods for inducing cells to express proteins using nucleic acids, methods, kits and devices for transfecting, gene editing, and reprogramming cells, and cells, organisms, and therapeutics produced using these methods, kits, and devices. Methods and products for altering the DNA sequence of a cell are described, as are methods and products for inducing cells to express proteins using synthetic RNA molecules. Therapeutics comprising nucleic acids encoding gene-editing proteins are also described.
Abstract: The present invention provides genetically engineered strains of Pichia capable of producing proteins with reduced glycosylation. In particular, the genetically engineered strains of the present invention are capable of expressing either or both of an ?-1,2-mannosidase and glucosidase II. The genetically engineered strains of the present invention can be further modified such that the OCH1 gene is disrupted. Methods of producing glycoproteins with reduced glycosylation using such genetically engineered stains of Pichia are also provided.
Type:
Grant
Filed:
January 10, 2014
Date of Patent:
June 7, 2016
Assignees:
VIB, VZW, Research Corporation Technologies, Inc., Universiteit Gent
Inventors:
Roland Contreras, Nico L.M. Callewaert, Steven C.J. Geysens
Abstract: Compositions and methods are provided for achieving persistent, high level expression of transgenes in vitro and in vivo. Aspects of the invention include vectors comprising an intronic cassette that comprises plasmid elements, and methods that rely on the use of vectors comprising an intronic cassette that comprises plasmid elements. These compositions and methods find use in many applications, including therapeutic applications such as in gene therapy; synthesis applications such as in the synthesis of peptides, proteins, and RNAs, e.g. for research or therapeutic purposes; and research applications, such as in the production of transgenic cells and animals. In addition, reagents, devices and kits thereof that find use in making the subject compositions and practicing the subject methods are provided.
Type:
Grant
Filed:
January 17, 2013
Date of Patent:
May 24, 2016
Assignee:
The Board of Trustees of the Leland Stanford Junior university
Abstract: The technology relates to a nucleic acid expression cassette comprising a TR element encoding an mRNA molecule that is translated in stressed and/or dying cells, and a nucleotide sequence operably linked to the TR element, that is a first open reading frame (ORF) sequence and encodes a polypeptide or a fragment thereof and is co-translated with the TR element. The technology further relates to mammalian cells and a transgenic animal comprising such expression cassette. Further included are kits comprising the expression cassette, and methods for determining toxicity, and killing a target cell.
Abstract: Stimulation of target cells using light, e.g., in vivo or in vitro, is implemented using a variety of methods and devices. One example involves a vector for delivering a light-activated molecule comprising a nucleic acid sequence that codes for light-activated molecule. The light-activated molecule includes a modification to a location near the all-trans retinal Schiff base, e.g., to extends the duration time of the open state. Other aspects and embodiments are directed to systems, methods, kits, compositions of matter and molecules for ion channels or pumps or for controlling currents in a cell (e.g., in vivo or in vitro environments).
Type:
Grant
Filed:
April 1, 2013
Date of Patent:
April 12, 2016
Assignee:
The Board of Trustees of the Leland Stanford Junior University
Inventors:
Karl Deisseroth, Ofer Yizhar, Lisa Gunaydin, Peter Hegemann, Andre Berndt
Abstract: The present invention is directed to constructs, compositions and methods for modulating platelet-type 12 lipoxygenase (12-LO) in adipose tissue in vivo. Specifically, the invention provides constructs encoding expression-inhibiting oligonucleic acids, e.g., antisense and RNA interfering (RNAi) molecules, targeted to a platelet-type 12-LO gene or a transcript thereof, which are capable of reducing or silencing platelet-type 12-LO expression specifically in adipocytes and pre-adipocytes. Vectors of these constructs (including, but not limited to, viral vectors), compositions of them and methods of using same for the treatment and amelioration of conditions associated with excess fat cell mass and obesity are also provided.
Type:
Grant
Filed:
November 1, 2007
Date of Patent:
March 8, 2016
Assignee:
The Medical Research Fund at the Tel-Aviv Sourasky Medical Center
Inventors:
Gary Weisinger, Rona Limor, Naftali Stern
Abstract: Bioimplants and methods of making the bioimplants are provided. The bioimplants comprise biological tissues having conjugated thereto adjunct molecules. The biological tissues are sterilized with a chemical sterilizing agent, such as a water soluble carbodiimide. The processes of making the bioimplants include a process in which an adjunct molecule is conjugated to a biological tissue during the sterilization process.
Type:
Grant
Filed:
June 30, 2010
Date of Patent:
March 1, 2016
Assignee:
Synovis Orthopedic and Woundcare, Inc.
Inventors:
Chandrasekaran Nataraj, Gregg Ritter, Thomas Sander