17 results about "Gene product" patented technology

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.

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.

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.

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.

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.

The present application relates to the field of biotechnology, in particular to a tobacco NtClpR4 gene and application. The present application clones a nicotine transformation related gene NtClpR4 from tobacco for the first time, which encodes an ATP-dependent tyrosine protease Clp related subunit, the amino acid sequence is shown as SEQ ID NO: 2, and the nucleotide sequence is shown as SEQ ID NO: 1; the gene product affects the synthesis of nornicotine. After mutation of the NtClpR4 gene, the nicotine content of cured tobacco leaves decreases, and the nornicotine content increases, which provides a new technical means for nicotine transformation regulation and tobacco breeding.

The present disclosure relates to the introduction of expression constructs encoding different gene products, such as proteins or nucleic acids, into a host cell line containing a plurality of docking sites for insertion of nucleic acid constructs in defined ratios, and the production of proteins requiring the expression of at least two gene products.

A genetically modified Salmonella cell (GMSC) engineered to exhibit specific targeting to cells and regulated delayed lysis in vivo, the GMSC comprising a first heterologous nucleic acid that encodes a first gene product that causes the GMSC to be selectively localized to and / or internalized by a target cell in vivo and a second heterologous nucleic acid that encodes a second gene product that facilitates killing of the target cells following internalization.

Provided are a capsid-modified adeno-associated virus (AAV), preparation therefor and a use thereof. Specifically, provided are an AAV capsid protein variant, a preparation method for recombinant AAV particles comprising a variant capsid protein, and compared with an AAV containing an unmodified parental AAV capsid protein, increased infectivity and gene product expression thereof in target cells such as retinal cells.

Compositions and methods discussed herein provide for treatment or prevention of a disease or disorder, or its symptoms, using a modified herpes simplex virus (mHSV) vector comprising at least two transgenes, wherein at least a first transgene encoding a gene-editing system, gene-deletion system, or bridge-editing system is expressed to knock out or delete an endogenous target gene and at least a second transgene encoding a corrected copy of the endogenous targeted gene, wherein the gene product of the second transgene replaces the gene product of the endogenous target gene.

Provided herein are clonal strains of a vaccinia virus that exhibits enhanced anti-tumor properties and / or reduced immunogenicity, and recombinant vaccinia virus derived from the same. Also provided herein are recombinant oncolytic virus strains that include an inactivating mutation in one or more viral genes, and / or one of more heterologous nucleic acids each encoding one or more heterologous gene products. The viruses, e.g., vaccinia viruses, provided herein, including recombinant vaccinia viruses, can be used as an oncolytic virus therapy, e.g., an oncolytic vaccinia virus therapy, for treating cancer. Also provided herein are pharmaceutical compositions and methods and uses of the viruses, e.g., vaccinia viruses, for treating cancer, as well as nucleic acids encoding the viruses.

Molecules for inhibiting arachidonate 15-lipoxygenase (ALOX-15) gene products including dsRNA (dsRNA) agents such as small interfering RNAs (siRNAs) for therapeutic use, additionally, methods to inhibit the expression of a target gene by administering these agents for the treatment of diseases involving ALOX-15 gene products.

This disclosure relates to Machado-Joseph disease (MJD) or spinocerebellar ataxia type 3 (SCA3), an autosomal dominant neurodegenerative disorder caused by excessive repetition of the polyglutamine-coding region in the ataxia protein-3 (ATXN3) gene. The extended ATXN3 readily aggregates and interferes with multiple cellular systems, ultimately leading to cellular dysfunction and death in specific neuronal populations. To date, no treatments have been developed that can reverse or delay the progression of MJD / SCA3. Strategies based on inhibiting harmful gene products have shown promising results in preclinical studies. However, these strategies do not target the root cause of the disease, producing incomplete and / or transient therapeutic effects in target cells or tissues. Recently, gene-based therapies, including the CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) system for gene editing, have been successfully used to permanently inactivate and correct disease-related genes, offering hope for the development of curative therapies for hereditary diseases.

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.

Adenovirus expression vectors and production methods and cell lines are provided. The adenovirus expression vectors can include: a) one or more mutations that render the adenovirus replication-incompetent; b) at least one nucleotide sequence encoding a protein or RNA. A method of synthesizing an adenovirus vector is also provided. The synthesis method can include: a) generating a plurality of overlapping adenovirus subfragments, each subfragment comprising a portion of the full genome of the adenovirus; b) circularizing the subfragments to form a plasmid structure; and c) assembling the circularized subfragments into a linear structure, wherein the vector comprises a combination of two or more subfragments. A mammalian cell line configured to replicate an adenovirus vector is also provided, wherein the cell line includes nucleotide sequences expressing E1A and E1B gene products, but does not contain other adenovirus sequences.

This invention relates to the field of biotechnology and provides a method for screening positive single B cells that secrete target substances and a method for preparing monoclonal antibodies. The positive single B cell screening method includes the following steps: isolating PBMCs from the peripheral blood of immunized animals; sorting memory B cells using magnetic beads and separation antibodies; activating and culturing memory B cells in vitro using an activation medium; and loading, analyzing, and exporting target positive cells from the activated memory B cells using a Beacon single-cell photoconductive system. This invention achieves high-throughput screening of FYL positive single B cells through the Beacon single-cell photoconductive system. Gene products are obtained through reverse transcription and PCR amplification, and accurate sequences are obtained through sequence analysis. The synthesized plasmid is expressed in a eukaryotic system, and high-purity FYL rabbit monoclonal antibodies are obtained through affinity chromatography, laying the foundation for the development of a rapid fentanyl detection kit.

A diazotrophic microbe is genetically modified to secrete a nitrogen-containing compound in an amount greater than a comparable control diazotrophic microbe. In one or more embodiments, the diazotrophic microbe is an endophyte. In one or more embodiments, the genetic modification includes a deletion or disruption of a gene involved in import or export of nitrogen-containing compounds. In some of these embodiments, the genetic modification includes a deletion or disruption of a gene encoding a gene product that transports ammonium into the cell, recycles lost ammonium back into the cell, and / or transports ammonia out of the cell. In one or more embodiments, the amount of secreted nitrogen-containing compound is effective to support the growth of a non-diazotroph in co-culture.