105 results about "Gene product" patented technology

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 present invention relates to compositions and methods for subpopulation-specific modulation of cell function and for treating repeat expansion disorders comprising genetic, degenerative, neurological and cellular diseases, including immune disorders. Also provided are research kits for subpopulation-specific modulation of protein activity and the corresponding potential to discover novel therapeutic compositions. More specifically, the disclosed compositions and methods selectively up- or downregulate at least a first population of a cellular protein or therapeutic target with minimal or negligible effect on the activity of at least a second population of the cellular protein or therapeutic target.

The present invention relates to a modified adeno-associated virus (AAV) capsid polypeptide and a novel recombinant adeno-associated virus (rAAV) comprising the modified AAV capsid polypeptide for delivering the gene product for the therapy. The present invention also relates to a pharmaceutical composition comprising the rAAV of the present invention, and a method of treating a retinal disease comprising the administration of the rAAV or the pharmaceutical composition of the present invention to an eye of a subject in need thereof.

The present invention relates to the field of therapy and drug delivery. More specifically, therapeutic methods and pharmaceutical compositions for treating disorders of blood-tissue barrier-protected organs that harbor substantial populations of postmittal neurons, such as organs derived from the neural tube, including the central nervous system and the eye. The disclosed methods and compositions involve topical administration of an effector component that targets intracellular biological targets to such organs, in combination with a saponin component that enhances the effective uptake of the effector component into cells and / or enhances the effective delivery of the effector component within cells where the biological target is present. For example, the effector component may be an oligonucleotide therapeutic that targets gene products associated with CNS and / or ocular disorders. Due to the cellular uptake stimulating and / or endosomal escape enhancing effects of the saponin component, the neuropharmaceuticals and ophthalmic compositions presented herein for topical administration to the CNS and / or eye, respectively, can be formulated with lower concentrations of the effector component and / or lower volumes, which provides safety benefits to neurons and patient comfort.

Plant cell fate and development is altered by treating cells with cellular reprogramming factors. Embryogenesis inducing embryogenesis factor genes and / or morphogenic developmental genes are used as cellular reprogramming factors, specifically comprising polypeptides or polynucleotides encoding gene products for generating doubled haploids or haploid plants from gametes. Maize microspores treated by contacting the isolated cells with an exogenous purified, recombinant embryogenesis inducing embryogenesis factor gene products and / or morphogenic developmental gene polypeptide results in embryogenesis. The gametes of a maize plant develop into embryoids when transformed with a genetic construct including regulatory elements and structural genes capable of acting in a cascading fashion to alter cellular fate of plant cells. Embryogenesis factor proteins and / or developmental morphogenic proteins expressed from a genetic construct are used for ex situ treatment methods and for in planta cellular reprogramming.

A method of direct transdifferentiation of somatic cells into other somatic cells may be convenient and still have good reproducibility, excellent production efficiency, and short performed time. Methods for direct transdifferentiation of somatic cells into other somatic cells may include: (a) introducing a GLIS family gene, a mutated GLIS family gene or a gene product thereof into somatic cells; and (b) culturing the gene-introduced somatic cells in a culture medium containing a component that induces differentiation of the somatic cells or precursor cells of the somatic cells into other somatic cells.

The application discloses a recombinant T cell with a knocked-out STARD7 gene and an application thereof, relates to the technical field of immune cells, and discloses that the recombinant T cell does not contain a functional STARD7 gene, or a biological function of a STARD7 gene product of the recombinant T cell is inhibited. In the application, the STARD7 is knocked out in T cells by means of CRISPR-Cas9 gene editing in an early stage of acute infection of mice, differentiation of SLECs is significantly damaged, and the proportion and quantity of MPECs are increased to a certain extent; therefore, the knockout of the STARD7 can be used as a treatment method for increasing the quantity of MPECs and regulating immune cells, so as to resist acute infection caused by external pathogens and promote the maintenance of memory cells existing in the body for a long time.

The application provides application of Brassica napus gene BnDTX19 in sclerotinia prevention and control, and is obtained by creating transgenic rapeseed to change the disease resistance of rapeseed material. The application constructs the super-expression and RNAi transgenic rapeseed of BnDTX19, firstly discloses the positive regulation function of the gene on sclerotinia resistance, provides the application of the BnDTX19 gene in obtaining high-sclerotinia-resistant rapeseed material by creating super-expression rapeseed, and the application of the BnDTX19-RNAi rapeseed in obtaining sclerotinia-resistant rapeseed material with weakened sclerotinia resistance. The BnDTX19 gene product provided by the application has the function of transporting the key pathogenic factor oxalic acid of sclerotinia to promote disease resistance, and is a new gene resource suitable for creating and breeding new sclerotinia-resistant rapeseed materials and new varieties.

Provided are methods for rapid multiplex HLA genotyping and transcript quantitation. In some embodiments, the presently disclosed methods include providing a. sample from the cell, tissue, or organ that has mRNA derived from an HLA gene product; reverse transcribing the mRNA to produce a. pool of cDNAs; amplifying the cDNAs that result from reverse transcription of HLA-specific mRNAs to produce a pool of HLA-specific cDNAs; and determining the sequence of each HLA-specific cDNAs, whereby the HLA status of the cell, tissue, organ, or subject is determined. Also provided are methods for genotyping donor cells, tissues, and / or organs meant for transplantation into recipients. In some embodiments, the methods can be used to identify the presence of absence of each of HLA-A, -B, -C, -DRB 1 / 3 / 4 / 5, -DQA1, and -DQB1 in a biological sample.

The present invention relates to the field of therapy and drug delivery. More specifically, disclosed herein are therapeutic methods and pharmaceutical compositions for treating disorders of blood tissue barrier shielded organs having a large number of populations of post-mitotic neurons, such as organs derived from neural tubes, including the central nervous system and the eye. The disclosed methods and compositions involve topically applying into such organs a combination of an effector component that targets a biological target in a cell, and a saponin component that promotes effective ingestion of the effector component into the cell and / or enhances effective path steering of the effector component within the cell in which the biological target is present. For example, the effector component may be an oligonucleotide therapeutic agent targeting gene products associated with CNS and / or disorders of the eye. Due to the cellular uptake stimulating effect and / or endosomal escape enhancing effect of the saponin component, the neuropharmaceutical and ophthalmic compositions presented herein for topical administration into the CNS and / or eye, respectively, may be formulated with lower concentrations of effector components and / or in smaller volumes, this brings safety benefits to the neurons and the comfort of the patient.

The present invention is in the field of viral immunotherapy. The invention provides 1) an efficient recombinant poxvirus expressing a toxic conditional gene product that affects the viability of the recombinant poxvirus itself if left unregulated, or 2) a recombinant poxvirus expressing a conditional gene product with an improved safety profile, a method of producing them, a composition comprising them and therapeutic uses related thereto.

Methods for the fermentative production of O-phosphoserine, cysteate, or taurine in microbes or unicellular organisms that contain a serB mutation that either decreases serB expression, reduces the amount of the serB gene product or results in a serB gene product with low enzymatic activity. Genetic modifications of the O-phosphoserine, cysteate, or taurine and / or substrate biosynthetic pathways in unicellular organisms that include bacteria, algae, microalgae, diatoms, yeast, or fungi are disclosed. Also disclosed are fermentation and processing methods for the production of various O-phosphoserine-, cysteate-, or taurine-containing products and the use of the cells, fermentation broth or extracts that contain O-phosphoserine, cysteate, or taurine to produce products for use in food, feed, beverages, dietary and health supplements, cosmetics, personal care, pharmaceuticals, agricultural production, or surfactants.

The present disclosure provides recombinant adeno-associated virus (AAV) virions with altered capsid protein, where the recombinant AAV (rAAV) virions exhibit greater infectivity of a central nervous system (CNS) cell, compared to wild-type rAAV, and where the rAAV virions comprise a heterologous nucleic acid. The present disclosure provides methods of delivering a gene product to a CNS cell in an individual.

The invention is characterized by a method and application of codon optimization of a gene product. Specifically, the present disclosure features a codon optimization method that promotes the increase in mRNA half-life and stability by reducing the frequency of m6A modification, thereby achieving the purpose of improving protein production. Additional methods of delivering codon optimized gene products are disclosed. The methods of the present disclosure are clinically related to gene and cell therapy as well as vaccine development.

The present invention provides the use of an antigen-binding molecule that binds to HER3 for the treatment or prevention of a HER3-related cancer wherein the HER3-related cancer does not comprise a genetic variation that results in an increase in MET expression or an increase in gene product activity.

Provided is a method for production by direct transdifferentiation of somatic cells into another somatic cells which is convenient, has good reproducibility, is excellent in production efficiency, and is performed in a short period of time. The method for production by direct transdifferentiation of somatic cells into another somatic cells comprises: (a) a step of introducing a GLIS family gene, a mutated GLIS family gene or a gene product thereof into somatic cells; and (b) a step of culturing the gene-introduced somatic cells in a culture medium containing a component that induces differentiation of the somatic cells or precursor cells of the somatic cells into another somatic cells.

Provided herein are methods of hypoimmunogenicity, such as bioengineering methodologies and materials, including hypoimmunogenicity (such as engineering hypoimmunogenicity) methodologies and materials useful in, for example, genetically modifying and / or otherwise altering at least one target gene or gene product, processes for producing engineered hypoimmunogenic cells, manufacturing of engineered hypoimmunogenic cellular compositions, and uses thereof.

Aspects of the disclosure relate to compositions and methods for delivering a transgene (e.g., a transgene encoding one or more gene products) to a target cell (e.g., a brain cell). The disclosure is based, in part, on adeno-associated virus (AAV) capsid proteins comprising one or more amino acid substitutions, and methods of using the same for delivery of a transgene to tissues and cells of the central nervous system (e.g., brain cells).

This disclosure provides replication-incompetent adenoviral vectors useful in vaccine development and gene therapy. The disclosed vectors comprise a selective deletion of E3 and are particularly useful for preparation of vaccines development and for gene therapy using toxic transgene products that result in vector instability that occurs when the entire E3 domain is deleted.

Provided herein are improved methods for transducing immune cells, such as T cells, with retroviral vectors to express exogenous gene products, such as chimeric antigen receptors (CARs). Provided herein are methods that increase transduction efficiency thereby increasing the percentage of immune cells in a population expressing the exogenous gene product. Associated cells, cell populations, compositions and methods of use are also provided.

Herein disclosed are compositions comprising synthetic chromosomes and methods of their use to treat diseases and disorders (e.g., cancers, genetic and autoimmune diseases). Specifically described are methods of constructing synthetic chromosome compositions bearing one or multiple genes as well as regulatory sequences that control expression of the gene(s) such that, when these are expressed from the synthetic chromosome in an animal cell, at least one medicinal gene product is reliably, faithfully and indefinitely produced by the animal cells. As an example, the presently disclosed compositions and methods are used to bioengineer cells to enable them to express the entire dystrophin gene and additional regulatory nucleic acid sequences under tightly controlled conditions, allowing the present methods and compositions to be used as cellular medicines for treatment of diseases such as muscular dystrophies.

This invention describes a transgenic non-human animal model for testing postnatal conditionally inducible plasmalogen deficiency. The genome of the animal model comprises genes capable of regulating the plasmalogen biosynthesis pathway, and these genes have regulatory regions. Within the regulatory regions are at least one conditionally inducible gene editing site that, when edited, blocks gene expression. The genome also has nucleic acid editing sequences that are integrated into a locus separate from the gene encoding the gene product. The gene product, when conditionally induced, edits the gene editing site, ultimately downregulating or disrupting the plasmalogen biosynthesis pathway. Methods and uses comprising the transgenic non-human animal model are also provided.

The invention provides compositions and methods for performing mammalian cell genetics, e.g., genetic screens, using near-haploid cells. The invention further provides genes and gene products isolated using the inventive methods and methods of use thereof.

Described herein are methods for obtaining enucleated cells from nucleated cells. Also described herein are methods for cell processing, including providing a composition containing nucleated cells and enucleating at least a portion of the nucleated cells to produce an enucleated cell fraction. Also described herein are methods for cell processing, including expressing the heterologous gene product. Also provided are pharmaceuticals compositions comprising an enucleated cell.

The present invention relates to oncolytic vaccinia viruses and recombinant viruses and methods of use thereof. In particular, the present invention provides a cloned vaccinia virus strain exhibiting enhanced anti-tumor properties and / or reduced immunogenicity, as well as a recombinant vaccinia virus derived therefrom. The present invention also provides recombinant oncolytic virus strains comprising an inactivated mutation in one or more viral genes, and / or one or more heterologous nucleic acids each encoding one or more heterologous gene products. The viruses (e.g., vaccinia viruses, including recombinant vaccinia viruses) provided herein are useful as oncolytic viral therapies (e.g., oncolytic vaccinia viral therapies) for the treatment of cancer. The invention also provides pharmaceutical compositions of the virus, such as vaccinia virus, and methods and uses of the virus, such as vaccinia virus, for the treatment of cancer, as well as nucleic acids encoding the virus.

Compositions and methods and for enhancing the resistance of plants to plant diseases caused by potyviruses are provided. The compositions comprise nucleic acid molecules encoding resistance (R) gene products and variants thereof and plants, seeds, and plant cells comprising such nucleic acid molecules. The methods for enhancing the resistance of a plant to plant disease caused by a potyvirus comprise introducing a nucleic acid molecule encoding an R gene product into a plant cell. Additionally provided are methods for using the plants in agriculture to limit plant disease.

Disclosed is a method and a device for determining whether a sample contains ampicillin-resistant (AMP-R) non-typhoid Salmonella (NTS). The method comprises detecting, in the sample, the presence of a combination of certain genes or their gene products, wherein the presence of said combination indicates the sample contains AMP-R NTS.

The present disclosure provides infectious recombinant adeno-associated virus (rAAV) virions that comprise a variant capsid protein and a heterologous nucleic acid. The present disclosure further provides the variant adeno-associated virus (AAV) capsid proteins (and / or a nucleic acid encoding the variant AAV capsid proteins), which confer to an infectious rAAV virion an increased resistance to human AAV neutralizing antibodies. The present disclosure further provides host cells comprising an infectious rAAV virion and / or a nucleic acid encoding a subject variant AAV capsid protein. The present disclosure further provides methods of delivering a heterologous nucleic acid to a target cell where the target cell is contacted with a subject infectious rAAV virion. The present disclosure further provides methods of delivering a gene product to an individual, the methods generally involving administering an effective amount of a subject rAAV virion to an individual in need thereof.

This disclosure describes the characteristics of the “energetic” cancer stem cell (e-CSC) phenotype. This distinct sub-population of cancer stem cells (CSCs) has a unique energetic profile compared to bulk CSCs, being more glycolitic, having higher mitochondrial mass and elevated oxidative metabolism. e-CSCs also show an increased capacity to undergo cell cycle progression, enhanced anchorage-independent growth, and ALDH-positivity. The e-CSC phenotype presents new targets for cancer therapeutics, and in particular the anti-oxidant response, mitochondrial energy production, and mitochondrial biogenesis of e-CSCs makes them highly susceptible to mitochondrial inhibitors that target e-CSC anti-oxidant response, mitochondrial energy production, and mitochondrial biogenesis. Gene products for e-CSCs are disclosed, as well as classes of mitochondrial inhibiting therapeutic agents. Also disclosed are methods for identifying and separating e-CSCs front bulk cell populations.

This invention relates to oncolytic vaccinia viruses, recombinant viruses thereof, and methods of use. Specifically, the invention provides vaccinia virus clone lines and recombinant vaccinia viruses derived therefrom, said vaccinia virus clone lines exhibiting enhanced antitumor properties and / or reduced immunogenicity. The invention also provides recombinant oncolytic virus lines comprising inactivating mutations in one or more viral genes, and / or one or more heterologous nucleic acids each encoding one or more heterologous gene products. The viruses provided by this invention (such as vaccinia viruses, including recombinant vaccinia viruses) can be used as oncolytic virus therapies (such as oncolytic vaccinia virus therapy) for the treatment of cancer. The invention also provides pharmaceutical compositions of viruses (such as vaccinia viruses), methods and uses of viruses (such as vaccinia viruses) for the treatment of cancer, and nucleic acids encoding said viruses.