101 results about "Genetic transfer" patented technology

The invention provides a grouper microinjection method, and belongs to the technical field of aquaculture. According to the method, grouper eggs are injected by utilizing a grouper microinjection needle, the microinjection needle is prepared by calcining a glass capillary tube by utilizing a needle pulling instrument, and the parameters of the needle pulling instrument are set as P = 500mW, HET = 720ms, PULL = 60, VEL = 20mu m / s and TIME = 250ms; the outer diameter of the glass capillary tube is 1.0 mm, the inner diameter of the glass capillary tube is 0.75 mm, and the length of the glass capillary tube is 10 cm. The method for injecting the grouper eggs with the micro-injection needle is simple and easy to operate, exogenous gene transfer and endogenous gene accurate editing of the grouper can be realized by adopting the method disclosed by the invention, the target gene editing efficiency is high, and powerful support is provided for deeply researching gene functions of the grouper and cultivating excellent culture varieties.

A method of correcting singletons in a selected AAV sequence in order to increasing the packaging yield, transduction efficiency, and / or gene transfer efficiency of the selected AAV is provided. This method involves altering one or more singletons in the parental AAV capsid to conform the singleton to the amino acid in the corresponding position(s) of the aligned functional AAV capsid sequences.

The present disclosure relates to compositions and methods for the treatment of cardiomyopathy. Several embodiments provided for herein relate to virally-mediated transfer of a gene to host cells to induce expression of an encoded polypeptide, protein or other product to host in order to ameliorate one or more symptoms of the cardiomyopathy. In several embodiments, the methods and compositions relate to recombinant adeno-associated virus particles encoding human TNNT2 in order to treat cardiomyopathies, including dilated cardiomyopathy or hypertrophic cardiomyopathy.

This product is a work cell for cell therapy manufacturing, automating the entire manufacturing process, including cell concentration, separation, activation, gene transfer using viral vectors or electroporation, expansion culture, filling, finishing, and formulation. This product can provide a self-contained cleanroom environment with a robotic work cell, all within the size of a conference room or a small pickup truck.

The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel elements that further improve expression. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

The present invention relates to a method for cultivating transgenic plants having increased anthocyanin content and enhanced tolerance to biotic and abiotic stress environments. The method comprises transferring a bHLH35 gene into recipient plants using a genetic engineering transformation method, to obtain transgenic plants having increased anthocyanin content and tolerance to biotic and abiotic stress environments. The bHLH35 gene may be selected from a ChbHLH35 gene from Corydalis hemidicentra or an AtbHLH35 gene from Arabidopsis thaliana.

The application discloses a Buster transposon system and application thereof, and the system comprises AnBT, CrBT, HvBT, LcBT and LlBT transposons and corresponding transposases, the nucleotide sequences of the AnBT, CrBT, HvBT, LcBT and LlBT transposons are shown in SEQ ID No. 1-5, and the transposases are wild type or enhanced type. The application obtains key elements such as target site repeat (TSD) and terminal inverted repeat (TIR) of the AnBT, CrBT, HvBT, LcBT and LlBT transposons and corresponding transposase sequences through molecular reconstitution, and constructs a set of gene transfer vector systems. The AnBT, CrBT, HvBT, LcBT and LlBT vector systems can effectively mediate gene transfer through cell verification, and have great application potential in transgenic animal preparation and gene therapy.

Tumor-specific T cell receptor (TCR) gene transfer enables specific and potent immune targeting of tumor antigens. The canonical cancer-testis antigen, NY-ESO-1, is not expressed in normal tissues but is aberrantly expressed across a broad array of cancer types. It has also been targeted with A2-restricted TCR gene therapy without adverse events or notable side effects. To enable the targeting of NY-ESO-1 in a broader array of HLA haplotypes, we isolated TCRs specific for NY-ESO-1 epitopes presented by four MHC molecules: HLA-A2, -B07, -B18, and -C03. Using these TCRs, we have developed an approach to extend TCR gene therapies targeting NY-ESO-1 to patient populations beyond those expressing HLA-A2.

The invention relates to a circular vector for plant transformation comprising: a first tethering nucleic acid and a second tethering nucleic acid; a nucleic acid encoding a polynucleotide of interest (POI); a nucleic acid comprising an origin of replication; and two or more nucleic acids encoding replicon proteins and methods for using the same. Also provided are plants produced by the methods of the invention and products produced from the plant.

A method for preparing a cell line for the amplification of a replication-defective recombinant virus, a defective virus and the use thereof, which belong to the technical field of genetic engineering. In the method for preparing the cell line for the amplification of the replication-defective recombinant virus, replication-related virus genes can be efficiently integrated into a cell genome by means of the gene transfer function of a transposase, and the expression of the replication-related genes is activated under the action of Cre recombinase, thereby activating the translation of corresponding proteins and ensuring the packaging and amplification of the replication-defective virus in cells, such that large-scale amplification of the replication-defective recombinant virus is realized. The cell line prepared by means of the provided method is suitable for the replication of a corresponding replication-defective recombinant virus, and provides a raw material basis for preparing a vaccine using a replication-defective recombinant virus as a candidate antigen and for preparing a gene therapy vector.

The invention discloses an erythromycin lactobionate raw material and a production process of erythromycin lactobionate for injection, which greatly increases the output value of Saccharopolyspora erythraea strains by improving a co-culture system and utilizing the synergistic metabolism between the two strains of Escherichia coli BL21 and Saccharopolyspora erythraea, and improves the yield of the Saccharopolyspora erythraea. Wherein a metabolite of the escherichia coli BL21 and peanut oil in a plate culture medium have a synergistic effect, and the growth time of a saccharopolyspora erythraea strain can be effectively shortened and the titer of erythromycin can be improved through a gene transfer effect. Meanwhile, the erythromycin lactobionate obtained through a microbe co-culture means can remarkably reduce the toxic effect on mouse livers, liver injuries caused by antibiotics are effectively relieved, and the erythromycin lactobionate is superior to traditional erythromycin lactobionate.

Disclosed are compositions and methods of treating muscular dystrophies, including Duchenne Muscular Dystrophy (DMD) through administration of fibroblasts and modified fibroblasts systemically and locally. In certain embodiments, fibroblast cells are utilized for replacement of dystrophin through fusion and / or other means of horizontal gene transfer. In other embodiments, the disclosure teaches the use of fibroblasts for reduction of inflammatory reactions and / or immunological reactions which propagate and enhance myodestructive aspects of Duchenne Muscular Dystrophy. In other embodiments, fibroblasts are utilized as vectors for gene therapy and / or gene modifications approaches.

The present disclosure relates to methods and materials involved in treating a mammal (e.g., a human) with cancer and / or infectious diseases. For example, the present disclosure provides a recombinant human cytomegalovirus (hCMV) vector that includes (e.g., is designed to include) a nucleic acid encoding a viral gene transfer vector genome (e.g., a heterologous viral gene transfer vector genome) and one or more nucleic acids encoding packaging polypeptides, such that a mammalian cell infected with the hCMV vector can produce and release a viral vector (e.g., an infectious lentiviral vector), which in turn can infect cells (e.g., immune cells) in vivo and, in some cases, drive expression of an exogenous polypeptide (e.g., a therapeutic polypeptide or an antigen receptor such as a chimeric antigen receptor (CAR)) in the infected immune cell in the mammal (e.g., a human) to induce an immune response in the mammal.

The present invention relates to a novel ionized lipid and a lipid nanoparticle composition using the same. The lipid nanoparticles comprising the novel ionized lipid according to the present invention exhibit excellent gene encapsulation rate and in vivo gene delivery rate, and as well as excellent gene delivery ability even upon re-administered, and thus can be advantageously utilized in the development of gene therapeutic agents for various indications.

The present invention provides a method for treating an auditory neuropathy spectrum disorder in a subject comprising transferring the gene of DFNB59 via an adeno-associated virus (AAV) vector to the subject.

The present invention refers to hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB). The invention further refers to corresponding nucleic acids producing these variants, to a gene transfer system for stably introducing nucleic acid(s) into the DNA of a cell by using these hyperactive variants of a transposase of the transposon system Sleeping Beauty (SB) and to transposons used in the inventive gene transfer system, comprising a nucleic acid sequence with flanking repeats (IRs and / or RSDs). Furthermore, applications of these transposase variants, the transpsoson, or the gene transfer system are also disclosed such as gene therapy, insertional mutagenesis, gene discovery (including genome mapping), mobilization of genes, library screening, or functional analysis of genomes in vivo and in vitro. Finally, pharmaceutical compositions and kits are also encompassed.

To provide a novel therapeutic approach to ornithine transcarbamylase deficiency.SOLUTION: The present application provides a modified adeno-associated virus vector that efficiently transfers genes to a liver in a living body (human, for example) by expressing ornithine transcarbamylase and reducing attack from neutralizing antibodies in blood. The present application provides a modified adeno-associated virus vector or the like, expressing ornithine transcarbamylase, comprising a modified VP1 protein including an amino acid sequence with at least one of position 472, position 587, and position 706 substituted with another amino acid in the amino acid sequence of VP1 protein, and not showing a cross-reaction with a neutralizing antibody to AAV2.SELECTED DRAWING: None

The present invention provides a method and apparatus for identifying horizontally transferred sequences from bacterial genomes in recipient insect genomes, relating to the field of biotechnology. The method provided by the present invention for identifying horizontally transferred sequences from bacterial genomes in recipient insect genomes more comprehensively and accurately identifies and analyzes fragments horizontally transferred from a certain type of bacteria to the genome of this species at the whole genome level; based on the horizontally transferred sequence fragments, horizontally transferred genes, gene fragments, and non-coding region fragments are further identified. This provides a comprehensive analysis of horizontal gene transfer between the genomes of a certain eukaryote and a certain type of bacteria, laying a solid, comprehensive, and reliable foundation for further research into the effects of horizontally transferred genes (fragments) on eukaryotes.

To provide a method for producing a γδT cell population which expresses functional TCR or CAR by preparing large quantities of γδT cells with high purity and efficiently introducing a foreign gene encoding TCR or CAR into the obtained γδT cells.SOLUTION: The present invention provides a method for efficiently producing highly pure γδT cells introduced with foreign genes by culturing in the presence of IL-7 and IL-15 after stimulating the γδT cells with a bisphosphonic acid ester derivative, followed by gene introduction. When a T cell receptor (TCR) or a chimeric antigen receptor (CAR) is introduced as the foreign gene, the γδT cells in which TCR and CAR are functionally expressed can be obtained.SELECTED DRAWING: None

This invention discloses a method for establishing a horizontal gene transfer system between wolfberry and tomato and obtaining transformed roots. Using tomato and wolfberry (both belonging to the Solanaceae family) as starting materials, the method reduces the lignin content of wolfberry cells, eliminates the isolation layer between distantly grafted cells, and connects the grafted cells of the two species. Adventitious roots are induced at the grafting site of the tomato scion by treating it with different auxins. Morphological and molecular biological studies demonstrate that horizontal gene transfer occurred between wolfberry and tomato cells mediated by grafting, and the induced adventitious roots are transformed roots from wolfberry to tomato. This invention establishes a technical system for efficient transfer of wolfberry genes to tomato and obtains transformed roots, achieving complementary advantages between herbaceous and woody plant genetic resources. Tomato plants with transformed root systems exhibit good root vigor, strong plant growth, and a long growth cycle. Obtaining transformed roots is of great significance for improving tomato yield, quality, and resistance.

An engineered transposable element, a gene transfer system comprising same, a method for using same and a kit. A disclosed composition, and the disclosed system and method can be used to insert heterologous nucleic acids into target nucleic acids in vitro or in cells.

The application discloses a method for normalizing and comparing horizontal gene transfer (HGT) network, comprising the following steps: S1, using Bayesian probability model to normalize HGT according to sequencing amount; S2, in the HGT network, each genome is represented as a vertex, and if HGT occurs between two genomes, there is an edge between the two vertices; S3, using different classification levels to annotate genome sequence, and obtaining HGT network of different classification levels; S4, analyzing HGT network through several topological properties of graph density, transitivity, homophily and algebraic connectivity. The graph density, transitivity, homophily and algebraic connectivity are used to describe the network, and the HGT network is compared among samples in different groups, so that a new analysis direction is provided for the correlation between metagenome and human phenotype, and the metagenome can be more systematically modeled and analyzed by calculating various topological properties to analyze the HGT network.

The invention provides compositions and methods for ameliorating defects in myosin VIIa (MYO7A) expression and / or function, including providing vectors for myosin VIIa expression and formulations comprising them, and methods of using them, for treating human retinitis pigmentosa (or retinal degeneration), and blindness and deafness such as that found in Usher syndrome. The invention provides in vivo gene therapy for ameliorating defects in myosin VIIa (MYO7A) expression and / or function, including compositions and methods for gene transfer of a human myosin VIIa (MYO7A) polynucleotide sequence that expresses multiple polypeptide isoforms of human myosin VIIa.

To provide nucleic acid sequences, expression vectors (e.g., vector genomes) and plasmids, compositions and viral vectors in which the nucleic acid encodes Factor IX (e.g. human Factor IX).SOLUTION: A nucleic acid sequence encodes human Factor IX protein, where the nucleic acid has a reduced number of CpG di-nucleotides compared to a wild-type sequence encoding human Factor IX.SELECTED DRAWING: None

The present invention provides polynucleotide vectors for high expression of heterologous genes. Some vectors further comprise novel transposons and transposases that further improve expression. Further disclosed are vectors that can be used in a gene transfer system for stably introducing nucleic acids into the DNA of a cell. The gene transfer systems can be used in methods, for example, gene expression, bioprocessing, gene therapy, insertional mutagenesis, or gene discovery.

This disclosure relates to adeno-associated virus variants capable of performing astrocyte-specific gene transfer. Administration of a recombinant viral vector containing nucleic acid encoding an AAV capsid protein mutant according to this disclosure enables the transfer and expression of therapeutic genes into specific astrocytes, thus demonstrating significant efficacy in the prevention or treatment of brain diseases.

The invention relates to a hybrid peptide consisting of amino acid sequences that bind nucleic acids on one side and cell membrane on the other side, in the fields of biology, genetics and chemistry. The invention particularly relates to a hybrid peptide developed to ensure the transfer of genes into the cell in the treatment of genetic diseases and incurable diseases such as some types of cancer.

The present disclosure relates to an adeno-associated virus vector capable of smooth muscle cell-specific gene delivery. With significantly enhanced tropism for smooth muscle cells, the adeno-associated virus variants of the present disclosure can be advantageously applied as a gene therapeutic agent for treating smooth muscle-related diseases.

The present invention relates to a method for efficiently expressing a target gene using a viral vector in immune cells, more specifically natural killer cells. By adding a simple process of freezing and thawing natural killer cells prior to transduction, the transduction efficiency of viral vectors can be dramatically improved without addition of enzymes or small molecules. The present invention is particularly useful for improving the efficiency of lentiviral vector that has a low gene transfer efficiency into natural killer cells, thereby obtaining natural killer cells in which desired genes are introduced to maximize their function, activity, and productivity as a source of immunotherapy.

Provided are cells that are resistant to mobile genetic elements or horizontal gene transfer, and methods for obtaining said cells. Also provided are methods for preventing the horizontal transfer of genetic information between a mobile genetic element and a first cell, cells making use of new genetic codons schemes and related subject matter, kits comprising mutually orthogonal cells, and mobile genetic elements. Also provided are methods of altering the susceptibility of a gene to mutations that alter the encoded amino acid sequence, methods for evolving or improving a protein, and methods for rendering a target gene more resistant to mutation. Additionally provided are uses of the cells for making polymers and methods comprising using the cells for making polymers.