245 results about "Open reading frame" patented technology

The present disclosure describes a new native peptide designated herein as Dwarf Open Reading Frame, or DWORF. This peptide enhances the apparent activity of the SERCA pump, is positively inotropic and lusitropic, and therefore is provided as a therapeutic agent for disorders characterized by cytosolic calcium overload.

The invention relates to a method for stimulating an immune response by intramuscular injection of an artificial nucleic acid molecule comprising an open reading frame encoding an antigen and a 3′-UTR comprising at least two poly(A) sequences. The method may yield an increased immune response to the antigen or an increased neutralizing antibody response to the antigen.

Synthetic RNA molecules comprising at least two RNA-binding protein (RBP)-binding motifs, wherein the at least two RBP-binding motifs bind the same first RBP and comprise non-identical sequences are provided. Synthetic RNA molecules comprising an RBP-binding motif that binds two orthogonal RBPs, comprising at least three RBP-binding motifs for three orthogonal RBPs or comprising a first RBP-binding motif, a second RBP-binding motif, a regulatory element and an open reading frame wherein the first and second RBP-binding motifs cooperatively enhance translation of the open reading frame are also provided. Compositions, cells and methods of use or generating the synthetic RNA molecules are also provided.

The invention relates to an artificial nucleic acid molecule comprising at least one 5′ UTR element which is derived from a TOP gene, at least one open reading frame, and preferably at least one histone stem-loop. Optionally the artificial nucleic acid molecule may further comprise, e.g. a poly(A)sequence, a poyladenylation signal, and / or a 3′ UTR. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and / or genetic vaccination.

Provided are a replicable RNA molecule and uses thereof, the present invention relates to a replicable RNA molecule comprising, from the 5'end to the 3 'end, a 5' cap, a 5 'UTR, an open reading frame encoding an RNA replicase, a promoter, a sequence of interest, a 3' UTR, and a poly (A) tail wherein the RNA replicase is capable of amplifying the replicable RNA molecule and an RNA molecule containing the sequence of interest and the 3 'UTR wherein the RNA replicase is derived from the group consisting of the group consisting of the MosdPedras virus, MDPV, EVEV, Riogro virus, Mycovirus, Mycovirus, Mycovirus, Mycovirus, Mycovirus, Mycovirus, Mycovirus, Mycovirus, Mycovirus, Mycovirus, Mycovirus and Mycovirus. RnV, Muslim virus, MUCV, Highland J virus, PIXV, TROV, Cabassu virus, TONV, Bebaru virus, BEBV, Formun virus, GETV, or Nnumu virus, and NDUV can be used in the treatment of a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, a disease, and a disease.

This disclosure provides improved RNA molecules, including mRNA molecules that can be produced by in vitro transcription and are suitable for in vivo transfection using an appropriate delivery vehicle, such as a lipid nanoparticle (LNP) or targeted lipid nanoparticle (tLNP). The improved RNA include particular combinations of 5' untranslated region (UTR) and 3' UTR, particular 3' UTRs, or particular open reading frame sequences. Also provided herein are compositions of the LNP, or tLNP with an antibody as a targeting moiety, such as anti-CD8 antibodies that are used as targeting moiety.

A CVB virus expression vector comprising a PIV5 W3A viral genome comprising a mutation at amino acid residue S157 or S156 of the P / V gene and a deletion of the small hydrophobic (SH) gene of the PIV5 W3A viral genome, wherein the amino acid substitution at amino acid residue S157 or S156 comprises a substitution of serine (S) with phenylalanine (F) or asparagine (N), and the SH gene has a deletion of the SH open reading frame or the entire SH gene transcription unit. The CVB virus expression vector expresses a heterologous polypeptide, including SARS-CoV-2 spike (S), and / or nucleocapsid (N) and / or membrane (M) proteins, RSV fusion protein (F), or other antigens.

The present invention generally relates to tRNA-based effector molecules for use in inserting missense mutations into the open reading frame (ORF) of a gene, e.g., for the treatment of repetitive sequence amplification diseases.

The invention provides a single-chain and double-protein-expressed circular RNA construct and a preparation method and application thereof, and the single-chain and double-protein-expressed circular RNA construct comprises 5'and 3 'introns used for realizing RNA self-splicing and cyclization as a first kind of introns; the 5'and 3 'homologous arms are used as pairing sites in RNA molecules; the exon 1 and the exon 2, which are connected after being spliced, can be spliced and connected together under the action of the first type of introns to form a complete and continuous open reading frame; an interval sequence for providing spatial and adjustment sequence structures; the IRES is used for starting translation; a glycine-serine linker as a flexible linker; a green fluorescent protein for gene expression monitoring; a luciferase for producing light by a catalytic chemical reaction. All the advantages of the double-IRES system for generating natural complete protein are reserved, and the core defect of low efficiency can be thoroughly overcome.

This invention provides a dengue / Zika virus genetically engineered vaccine and its application. The dengue / Zika virus vaccine comprises an open reading frame encoding envelope protein domain III (EDIII) and the non-structural protein NS1, and displays EDIII monomers in the delivery vector shell. Immunization with this vaccine primarily induces a type-specific antibody response, reducing the production of cross-antibodies and thus effectively avoiding or eliminating the risk of antibody-dependent enhancement of infection (ADE). This vaccine can be used to prevent dengue virus and Zika virus infection.

The present invention provides a pharmaceutical composition for treating cancer, the pharmaceutical composition comprising a nucleic acid that suppresses the expression of Chromosome 11 Open Reading Frame 97 (C11orf97). With the present invention, it becomes possible to treat cancer.

Disclosed herein are a messenger RNA (mRNA) comprising an open reading frame (ORF) encoding a protein of interest, a heterologous 5' untranslated region (UTR) and / or a heterologous 3' UTR for highly expressing the protein of interest. A method for synthesizing the mRNA and uses of the mRNA are also provided.

This invention relates to polynucleotides comprising UBE3A open reading frame (ORF) sequences, vectors comprising the same, and methods of using the same for delivery of the ORF to a cell or a subject and to treat disorders associated with aberrant expression of a UBE3A gene or aberrant activity of a UBE3A gene product in the subject, such as Angelman Syndrome.

The invention relates to the field of biology, and particularly provides a replication-deficient influenza virus vaccine skeleton which comprises a PA gene, an HA gene, an NP gene, an NA gene, an M gene, an NS gene and a PB2 gene of an influenza virus. The PB2 gene is replaced into a PB1 gene coding region of a vaccine skeleton; the open reading frame of the original PB2 gene can be replaced by an exogenous gene, so that the exogenous gene is inserted into the vaccine skeleton and expressed. The skeleton has the advantages that the capacity is large, the antigen can be efficiently and stably expressed, the passage is stable, and the like. Meanwhile, the invention also discloses a replication-defective influenza virus as well as a preparation method and application thereof.

Described herein are methods and compositions for accurate detection of cancer using ultrasensitive immunoassays, e.g., digital ELISA, to detect open reading frame 1 protein (ORF1p), which is encoded by the LINE-1 retrotransposon, in biofluids.

The invention relates to the technical field of bioinformatics and microbial genome analysis, in particular to a food-borne pathogenic bacterium typing method based on a wgMLST technology, which comprises the following steps of: preliminarily comparing a genome sequence with a constructed lightweight wgMLST database, and aiming at imperfectly matched gene loci in a comparison result, identifying the genomic sequence in the lightweight wgMLST database and identifying the genomic sequence in the lightweight wgMLST database. And expanding or cutting off the sequence according to the comparison region, predicting and extracting a potential open reading frame (ORF) sequence, and carrying out dictionary comparison on the potential ORF sequence until matching succeeds, thereby accurately judging the corresponding number of the allele. And traversing all imperfect matching sites and repeating the process to finally obtain a complete wgMLST typing spectrum. According to the method, a wgMLST typing task of large-scale high-throughput sequencing data can be efficiently processed; the complete analysis speed of single pathogenic bacteria is increased by nearly 10 times; a complete allele dictionary is introduced for secondary comparison and error correction, and it is ensured that imperfect matching site typing is correct and high in accuracy; and a technical support is provided for high-throughput strain detection and typing.

A lipid nanoparticle composition for use in the delivery of a self-amplifying RNA (saRNA) construct in one or more target cells is disclosed. The lipid nanoparticle composition comprises a lipid mixture comprising at least one (ionizable) cationic lipid, at least one helper lipid, a sterol, a corticosteroid such as Dexamethasone, and a least one lipid-polyethylene glycol conjugate. The saRNA construct comprises a first open reading frame which encodes one or more non-structural proteins, and a second open reading frame operatively linked to the first open reading frame. The second open reading frame comprises a coding region which encodes one or more target proteins. In some embodiments, the target proteins comprise one or more therapeutic proteins. In some embodiments, the target proteins comprise one or more one or more Cas proteins and / or variants thereof for use in CRISPR-based gene editing.

A method of producing a population of mature human pluripotent stem cell-derived sensory neurons (hPSC-SNs) expressing a target gene associated with at least one of nociceptive pain, chronic pain, pruriception and a nociceptive- or pruriceptive- mediated condition, the method comprising introducing into a population of human pluripotent stem cells (hPSCs) a composition comprising at least one site-directed nuclease targeting a site within the target gene, and at least one nucleic acid comprising a nucleotide sequence encoding at least one screenable, selectable marker that is flanked by (i) a nucleotide sequence homologous with a region located upstream of the target site within the target gene and (ii) a nucleotide sequence homologous with a region located downstream of the target site within the target gene, wherein the target site is located downstream of the open reading frame of the target gene, and wherein the site-directed nuclease cleaves the target site of the target gene and the nucleic acid encoding the screenable, selectable marker is inserted at the target site; covering said population of hPSCs under an extracellular matrix comprising at least one neuronal differentiation driver to produce a sensory committed neural crest population; contacting said sensory committed neural crest population with at least one neuronal differentiation driver and at least one neurotrophic factor to produce a population of early sensory neurons (SNs); and contacting said population of early SNs with at least one neurotrophic factor to produce a population of mature hPSC-SNs expressing at least one of SN marker or one pan neuronal marker; isolating the cells expressing the at least one screenable, selectable marker, wherein said population of mature hPSC-SNs expressed said one target gene associated with nociceptive pain, chronic pain, pruriception a nociceptive- or pruriceptive- mediated condition, and wherein said population of mature hPSC-SNs responds to a nociceptive and pruriceptive stimulus.

The invention provides a novel recombinant orf virus, the recombinant orf virus expresses interleukin 15, the recombinant orf virus is an ORFV (Open Reading Frame Virus) ORFs 005-008 gene deleted strain (marked as ORFV delta), and the IL15 gene expressed by the recombinant orf virus is one of a mouse source, a human source, a cat source or a dog source. According to the invention, an ORFV oncolytic virus is used as a carrier to develop a tumor immunotherapy drug ORFVdelta-IL15 expressing IL15, and the IL15 is expressed in tumor cells by using the characteristics of virus infection, so that the activation of NK cells and T cells in a tumor microenvironment is promoted while the tumor cells are cracked, and the anti-tumor effect of the oncolytic virus is enhanced. The invention provides a new strategy for tumor treatment.

The present disclosure provides compositions and methods for modulating C9orf72 gene transcription in patients in need thereof, including patients suffering from C9orf72 related diseases, such as amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD).

The present invention refers to a composition comprising Cutibacterium acnes (C. acnes) bacterial strains, wherein said strains are characterized by being modified to secrete a heterologous protein by insertion of an expression construct comprising at least one or more transcription promoter sequences, one or more ORFs (Open Reading Frames) encoding the heterologous protein, and one or more transcription termination sequences; wherein the heterologous protein is characterized by comprising at the N-terminus of the protein sequence, a secretion signal which is processed by C. acnes to secrete the protein, and wherein the heterologous protein is the superoxide dismutase enzyme.

Provided is a poly mRNA, comprising a plurality of mRNA structures. Each mRNA structure comprises: a cap region at the 5' end; and an open reading frame (ORF) encoding a polypeptide. The plurality of mRNA structures comprise a first mRNA structure and a second mRNA structure. The 3' terminus of the first mRNA structure is covalently linked to the second mRNA structure.

The present application relates to an isolated nucleic acid comprising (i) an open reading frame encoding a protein that is not to be expressed in a specific cell but to be expressed in a cell differing from the specific cell and (ii) a miRNA target site, wherein the miRNA target site is recognized by a miRNA that is expressed in the specific cell but substantially not expressed in the cell differing from the specific cell. The present application further relates to a particle comprising the afore-mentioned isolated nucleic acid as well as a pharmaceutical composition comprising the afore-mentioned particle. The present application is also concerned with a method of expressing a protein in a cell substantially not expressing a miRNA but not expressing the protein in a cell expressing said miRNA, either in vitro or in vivo in a subject, the use of the afore-mentioned nucleic acid, particle and pharmaceutical composition to express a protein in a cell-specific manner; with a method of treating a B cell cancer, an immune cell and a kit related to the afore-mentioned aspects.

This invention provides an organ fibrosis-related micropeptide, OFMP, and its applications. This type of micropeptide, OFMP, is a translational product with endogenous biological activity and stable existence, encoded by an open reading frame (ORF) in a long non-coding RNA. It is the first time that the long non-coding RNA encoding the micropeptide has been found to be significantly related to organ fibrosis and can encode the micropeptide. Synthetic peptides prepared based on the aforementioned OFMP micropeptide show significant inhibitory effects on several key indicators of organ fibrosis, including type I collagen α1 chain gene, α-smooth muscle actin, transforming growth factor β1, and connective tissue growth factor. This indicates that the OFMP micropeptide, the nucleotide sequence (ORF) encoding the micropeptide, and the long non-coding RNA sequence containing the nucleotide sequence have important clinical diagnostic and therapeutic value for organ fibrosis.

The invention relates to the technical field of genetic engineering, in particular to a small yellow croaker IRE1 alpha protein polyclonal antibody as well as a preparation method and application thereof. The invention provides a preparation method of a small yellow croaker IRE1alpha protein polyclonal antibody. The preparation method comprises the following steps: cloning an open reading frame of a small yellow croaker IRE1alpha gene, and constructing a small yellow croaker IRE1alpha gene recombinant expression vector pET-30a (+)-IRE1alpha. Performing inducible expression and purification on the recombinant protein of the Larimichthys polyactis IRE1 alpha, and performing specific analysis on Western Blot; and preparing the polyclonal antibody of the small yellow croaker IRE1 alpha. According to the invention, a recombinant expression vector of the small yellow croaker IRE1alpha gene is constructed by adopting molecular biology and genetic engineering methods, purified recombinant protein is obtained through induced expression and affinity chromatography, the prepared polyclonal antibody can be used for expression detection of the small yellow croaker IRE1alpha protein, and an important material foundation is laid for research on functions and action mechanisms of the small yellow croaker IRE1alpha protein.

The application relates to a high-throughput identification method of lncRNA coding peptides and application thereof, and belongs to the technical field of biology. The method combines a translationomics technology and a protein mass spectrometry technology, takes ribosome footprint sequencing data as a starting point, determines an open reading frame located on an lncRNA gene according to a three-base periodicity rule of a sequencing fragment, further constructs a proteogenomics index after systematic filtration, uses a computational proteomics method to detect an lncRNA translation event, and systematically identifies lncRNA coding peptides on the basis. The method has the characteristics of modularity and strong expandability, can be widely used for various types of proteome data, and can high-throughput identify and quantitatively detect lncRNA coding peptides. The application provides a prediction of molecular typing and prognosis of cancer patients based on an lncRNA coding peptide network model, and provides an important reference basis for precise medication of clinical patients.

This invention discloses a micropeptide, its encoding nucleic acid, and its application in regulating inflammatory activation of microglia. The amino acid sequence of the micropeptide is shown in SEQ ID NO:3. Simultaneously, this invention discloses the nucleic acid encoding the micropeptide, which contains either the nucleotide sequence shown in SEQ ID NO:1 or the nucleotide sequence shown in SEQ ID NO:2, wherein SEQ ID NO:2 is a small open reading frame located within the sequence of SEQ ID NO:1. This invention validated the translational expression of the micropeptide in microglia by constructing an EGFP reporter gene fusion vector and a SUMO tag fusion expression vector. Functional experiments showed that under oxygen-glucose deprivation / reperfusion injury conditions, the micropeptide significantly inhibited inflammatory activation of microglia. This invention provides a new potential target and candidate drug for the treatment of neuroinflammatory diseases such as stroke.

Nucleic acid molecules comprising at least one transcription regulatory element operably linked to an open reading frame, wherein the open reading frame encodes a single RNA transcript encoding GCaMP7f, a ribosomal skipping peptide, and a fusion protein of a nuclear protein and photoactivatable red fluorescent protein are provided. Expression vectors and cells comprising the nucleic acid molecules are also provided, as are methods of using the nucleic acid molecules for simultaneous labeling and measuring calcium and analyzing a target cell.

The invention discloses a nucleic acid molecule which comprises an open reading frame for coding an EBV antigen, and the EBV antigen is a specific antigen which is contained in a diseased cell of an EBV related disease, is coded by an EBV virus genome and is in a cell and on a cell membrane; the EBV antigen comprises LMP1, LMP2A, LMP2B, EBNA-LP, EBNA1, EBNA2, EBNA3A, EBNA3B, EBNA3C, EBNA4, EBNA5, EBNA6, EBNA7 and EBNA8, as well as lipid nanoparticles containing nucleic acid molecules, a pharmaceutical composition and application thereof, and belongs to the technical field of biology. The nucleic acid molecule, the mRNA, the composition containing the nucleic acid molecule, and the lipid nanoparticles can significantly increase the number of tumor specific immune cells, effectively activate immune response, and significantly inhibit tumor growth, and have significant anti-tumor activity for nasopharynx cancer and lymphoma.

The present application relates to a replicable RNA molecule and the use thereof. The replicable RNA molecule comprises, from the 5′ end to the 3′ end, a 5′ cap, a 5′ UTR, an open reading frame encoding an RNA replicase, a promoter, a sequence of interest, a 3′ UTR and a poly(A) tail, wherein the RNA replicase is capable of amplifying the replicable RNA molecule and is capable of amplifying an RNA molecule containing the sequence of interest and 3′ UTR, wherein the RNA replicase is a nonstructural protein or a functional variant thereof derived from Mosso das Pedras virus (MDPV), Everglades virus (EVEV), Rio Negro virus (RNV), Mucambo virus (MUCV), Highlands J virus (HJV), Pixuna virus (PIXV), Trocara virus (TROV), Cabassou virus (CABV), Tonate virus (TONV), Bebaru virus (BEBV), Fort Morgan virus (FMV), Getah virus (GETV) or Ndumu virus (NDUV).