Patent classifications
C12N2800/22
GENERATION OF CELL-BASED PRODUCTS FOR CONSUMPTION THAT COMPRISE PROTEINS FROM EXOTIC, ENDANGERED, AND EXTINCT SPECIES
The present disclosure relates to methods of preparing cell-based products for consumption that comprise proteins from exotic, endangered, and extinct species, as well as cell-based products for consumption.
NOVEL CRISPR ENZYMES AND SYSTEMS
Embodiments disclosed herein are directed to engineered CRISPR-Cas effector proteins that comprise at least one modification compared to an unmodified CRISPR-Cas effector protein that enhances binding of the of the CRISPR complex to the binding site and/or alters editing preference as compared to wild type. In certain example embodiments, the CRISPR-Cas effector protein is a Type V effector protein. In certain other example embodiments, the Type V effector protein is Cpf1. Embodiments disclosed herein are directed to viral vectors for delivery of CRISPR-Cas effector proteins, including Cpf1. In certain example embodiments, the vectors are designed so as to allow packaging of the CRISPR-Cas effector protein within a single vector. There is also an increased interest in the design of compact promoters for packing and thus expressing larger transgenes for targeted delivery and tissue-specificity. Thus, in another aspect certain embodiments disclosed herein are directed to delivery vectors, constructs, and methods of delivering larger genes for systemic delivery.
RECOMBINANT VECTOR CONTAINING IMMUNOGENIC PROTEIN OF AFRICAN SWINE FEVER VIRUS, RECOMBINANT BACTERIA AND USE THEREOF
A recombinant vector containing the immunogenic protein of African swine fever virus, a recombinant bacterium and use thereof, and relates to the technical field of gene recombination. The recombinant vector can be used to construct a recombinant Lactobacillus expressing the immunogenic protein of African swine fever virus, and after mixing the Lactobacillus solution that can secrete protein p72 and protein p54, respectively, an oral live bacterial preparation for preventing African swine fever can be prepared. The oral live bacteria preparation prepared by the disclosure can safely, effectively and quickly prevent the infection of African swine fever virus to pigs, and does not contain an immune process.
COMPOSITIONS FOR TREATING AND/OR PREVENTING CORONAVIRUS INFECTIONS
The disclosure provides recombinant vesicular stomatitis vims (VSV) particles, wherein the VSV glycoprotein (G) is replaced by a coronavirus spike (S) glycoprotein, or a fragment or a derivative thereof, as well as compositions, vaccines, kits, and methods for using the recombinant VSV particles. In a specific embodiment, the S glycoprotein is derived from Severe Acute Respiratory Syndrome coronavirus 2 (SARS-CoV-2) and the methods are for the treatment or prevention of a disease or disorder in a subject infected with SARS-CoV-2. In certain embodiments, the disease or disorder is COVID-19.
AAV-NAGLU VECTORS FOR TREATMENT OF MUCOPOLYSACCHARIDOSIS IIIB
This invention relates to viral vectors for delivery of α-N-acetylglucosaminidase (NAGLU) to a subject. In some aspects the NAGLU sequence is optimized for expression in human cells. The invention further relates to methods of using the vector to increase secretion of NAGLU from a cell and for treatment and prevention of mucopolysaccharidosis IIIB.
EXPRESSION OF MODIFIED PROTEINS IN A PEROXISOME
Disclosed herein include methods and compositions for making proteins in peroxisomes as well as methods of making cells for producing proteins in peroxisomes. Also disclosed herein are cells for producing a protein in a peroxisome, and methods for producing a protein in a eukaryotic cell containing a peroxisome as described herein.
ACTIVE DNA TRANSPOSON SYSTEMS AND METHODS FOR USE THEREOF
Provided are engineered transposable elements, gene transfer systems comprising the engineered transposable elements, as well as methods and kits for using the same. The compositions, systems, and methods are useful for inserting a heterologous nucleic acid into a target nucleic acid in vitro or in a cell.
Lentiviral vectors for delivery of <i>PKLR </i>to treat pyruvate kinase deficiency
The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells to provide gene therapy for pyruvate kinase deficiency.
RECOMBINANT VECTOR COMPRISING CODON-OPTIMIZED TIF1# POLYNUCLEOTIDE, AND USE THEREOF
Provided is a polynucleotide in which an N-terminal region of TIF1y gene is codon-optimized, a recombinant vector including the polynucleotide, and a use thereof.
GENE EXPRESSION SYSTEM USING STEALTHY RNA, AND GENE INTRODUCTION/EXPRESSION VECTOR INCLUDING SAID RNA
Simultaneous expression of a plurality of foreign genes by using a stealthy RNA gene expression system that is a complex that does not activate the innate immune mechanism and is formed from an RNA-dependent RNA polymerase, a single-strand RNA binding protein, and negative-sense single-strand RNAs including the following (1) to (8): (1) a target RNA sequence that codes for any protein or functional RNA; (2) an RNA sequence forming a noncoding region and derived from mRNA; (3) a transcription initiation signal sequence recognized by the RNA-dependent RNA polymerase; (4) a transcription termination signal sequence recognized by the polymerase; (5) an RNA sequence containing a replication origin recognized by the polymerase; (6) an RNA sequence that codes for the polymerase; (7) an RNA sequence that codes for a protein for regulating the activity of the polymerase; and (8) an RNA sequence that codes for the single-strand RNA binding protein.