133 results about "Viral vector" patented technology

Provided herein are immortalized mesenchymal stem cells comprising a replicating recombinant retrovirus. Methods for treating a cell proliferative disorder using these immortalized mesenchymal stem cells are also provided.

Disclosed are methods and compositions relating to antisense therapy for treating epilepsy, such as a focal epilepsy and temporal lobe epilepsy, in a subject in need thereof by targeting GRIK2 mRNA. In particular, the disclosure provides methods for treating symptoms (e.g., seizures) of epilepsy in a subject by administering a particular dose in a defined volume and in a specific route of administration of an inhibitory ribopolynucleotide or adeno-associated viral vector encoding the same, which is capable of inhibiting expression of GRIK2.

The present disclosure generally relates to a viral vector carrying a synthetic cancer antigen and a chemokine. Also provided herein are compositions and uses of the viral vector for delivering, such as tagging, a tumor with the synthetic cancer antigen.

The present invention relates to an adeno-associated virus (AAV) vector comprising in the 5' to 3' direction: a first AAV2 ITR sequence; a CMV enhancer sequence; a chicken β-actin promoter sequence; a nucleic acid sequence encoding an apolipoprotein 2 (APOE2) polypeptide comprising a Christchurch mutation; a polyA sequence; and a second AAV2 ITR sequence.

Provided herein are pharmaceutical compositions comprising a recombinant adeno-associated virus (AAV), a salt excipient or buffer, a sugar, and a surfactant. Also provided herein are methods for treating or preventing a disease in a subject in need thereof by administering to the subject a therapeutically effective amount of the pharmaceutical compositions.

The application discloses a liver-targeted gene editing system based on endogenous promoter hijacking and application, and belongs to the field of biological medicine. The system is composed of an LNP-wrapped modified Cas nuclease mRNA (first component) and a promoter-free viral vector carrying a therapeutic transgene donor (second component). The system uses LNP to realize the transient burst expression of Cas nuclease in the liver, mediates the generation of double-strand breaks at the site of endogenous high-expression genes, induces the site-specific integration of therapeutic transgenes without exogenous promoters, and hijacks the expression driven by endogenous promoters by using the splice acceptor (SA) mechanism. The application solves the risk of carcinogenesis caused by random integration of exogenous strong promoters and the immunotoxicity of long-term expression of nucleases through a "double safety lock" design. Experimental results prove that the system has high editing efficiency, long-term stability and no off-target, and can be used for various liver-derived metabolic diseases such as hemophilia, hypercholesterolemia and the like.

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.

Provided is an AAV vector capable of expressing a human Mef2c gene in the brain. Specifically, provided are an adeno-associated virus vector capable of expressing a full-length MEF2C protein, and the use thereof. The adeno-associated virus vector can significantly ameliorate the social deficit symptoms of mice with Mef2c gene deletion.

The present invention relates to: an AAV-based PDE6B viral vector for treating retinitis pigmentosa, the AAV-based PDE6B viral vector containing a tissue-specifically expressed PDE6A promoter; and a use of thereof, and provides a gene therapy for treating retinitis pigmentosa caused by PDE6B gene deficiency. The in vivo therapeutic efficacy of seven types of AAV5-PDE6B vectors was confirmed using an AAV by using a PDE6A promoter that is tissue-specifically expressed in photoreceptor rod cells that develop retinitis pigmentosa. An AAV5-PDE6A-450-PDE6B vector was selected as a candidate due to exhibiting strong tissue-specific expression in photoreceptor rod cells under even off-target conditions, unlike the gene expression characteristics of an AAV5-CMV-PDE6B vector, and was tested so as to be usable in the development of a gene therapeutic agent for treating PDE6B-deficient retinitis pigmentosa patients. Therefore, the present invention, related to AAV5-PDE6B for retinitis pigmentosa treatment and containing a tissue-specifically expressed PDE6A promoter, provides retinitis pigmentosa patients with an important treatment option having improved safety, and can be expected to have fundamental therapeutic effects compared to conventional treatments.

Herein is reported a method for separating recombinant viral particles from other compounds of a mammalian cell culture broth comprising the steps of adding a silica-based filter aid like diatomaceous earth (DE) or synthetic silica-based filter aid to a mammalian cell culture broth comprising recombinant viral particles at a weight ratio of more than 1:2 DE or synthetic silica-based filter aid / biological wet mass (BWM) to obtain a pre-filtration mixture and subjecting the pre-filtration mixture to alluvial filtration, whereby recombinant viral particles are separated from other compounds of the mammalian cell culture broth.

This provides methods for treating coronavirus disease 2019 (COVID-19). [Solution] This disclosure provides a chimeric adenovirus vector comprising nucleic acids encoding coronavirus disease 2019 (COVID-19) protein and an adjuvant, and a method of using the vector to induce an immune response to the SARS-CoV-2 protein for the purpose of treating COVID-19.

This invention provides improved compositions and methods for mRNA display libraries for the rapid production of stable recombinant high-affinity binders. [Solution] A composition, method, and use of recombinant viruses and / or recombinant viral vectors encoding distinct antibodies or antibody fragments prepared from a highly diverse nucleic acid library are disclosed. Preferably, the recombinant virus is a genetically modified low immunogenic virus, for example, an E2b deletion adenovirus. The highly diverse nucleic acid library is (1)V H -CDR1 / 2 sublibrary, (2) multiple V H -CDR3 sublibrary and (3)V L It includes or is derived from sublibraries, each of which includes multiple members. Preferably, each member of the sublibrary includes at least one random cassette having multiple degenerate base positions.

This invention relates to plant genetic engineering, and more particularly to a variegated Phalaenopsis orchid and its preparation method, the preparation method comprising the following steps: (1) according to the target gene with the base sequence shown in SEQ ID No. 1 PePDS (1) Synthesize specific nucleotide fragments; (2) Construct specific nucleotide fragments into a plant virus vector; (3) Introduce the recombinant plant virus vector obtained in step (2) into Phalaenopsis orchid plant material; (4) Screen to obtain Phalaenopsis orchids with silenced target genes. This invention achieves rapid, efficient, and non-transgenic leaf color variegation creation in commercial Phalaenopsis orchid varieties for the first time through VIGS technology; it not only breaks through the technical bottleneck of difficult and long cycle of genetic manipulation of orchid plants, significantly shortening the trait verification cycle, but also induces diverse and highly ornamental variegated leaf phenotypes such as albino, yellow, or purple in different varieties, and has the advantages of simple operation, low cost, and industrialization feasibility.

The present application relates to the use of an ANKRD50 gene inhibitor for the preparation of a medicament for the treatment of cancer. The present application also relates to nucleic acid molecules, nucleic acid constructs, viral vectors, viral particles for use as ANKRD50 gene inhibitors, and to pharmaceutical compositions and combinations for the treatment of cancer.

This invention relates to nucleic acids that encode and express full-length rod-derived cone survival factor (RdCVF) and human IgK signaling sequences, as well as viral vectors containing these nucleic acids. The invention also relates to compositions and pharmaceutical preparations containing these nucleic acids or vectors, methods for producing or secreting full-length RdCVF and human IgK signaling sequences, and therapeutic methods.

Recombinant polynucleotides encoding fragments of a human dysferlin protein are described herein. In addition, plasmids, viral vectors, dual vector systems, cells, and compositions comprising such recombinant polynucleotides are further described. Such recombinant polynucleotides, plasmids, viral vectors, dual vector systems, cells, and compositions may be used to treat dysferlinopathies. 1004798017 ABSTRACT2023206111 18 Jul 2023 1004798017 ABSTRACT2023206111 18 Jul 2023

Provided are compositions and methods for reducing pre-existing antibodies against viral vector or gene-editing related proteins. The compositions showed reduction in various antibody titers by administration of a liposome composition complexed with a protein or fragment thereof of a viral vector. The liposomes comprise phosphatidylcholine and phosphatidylserine, wherein some or all of the PS is present as lyso-PS. The compositions and methods can be used in conjunction with gene therapy and nucleic acid based vector based vaccinations and therapeutics.

This invention describes compositions and methods for the delivery of fully human post-translationally modified therapeutic monoclonal antibodies, or their antigen-binding fragments, that bind to C5, to human subjects for ocular indications, particularly for the treatment of AMD. The nucleotide sequence encoding the antibody is delivered via an rAAV vector that targets ocular tissue cells for transgene expression.

The present invention provides a temperature-sensitive negative-strand RNA virus or virus vector and an RNA genome thereof. According to the invention, provided is a negative-strand RNA virus or virus vector having a negative-strand RNA genome wherein the phosphoprotein (P protein) on the RNA genome has an amino acid mutation(s) corresponding to a substitution(s) in an amino acid(s) of the P protein corresponding to one or more or all of D433, R434, and K437 and optionally further has an amino acid mutation corresponding to a further amino acid substitution in an amino acid of the P protein corresponding to L511.

The application discloses a kind of modular serum-free cell culture medium and its application, belong to biomedical technology field.The culture medium includes: epidermal growth factor, recombinant insulin, cholesterol-phospholipid nanoparticles, beta-mercaptoethanol, pluronic F-68, glucose / glutamine, trace element mixed solution.The modular serum-free cell culture medium provided by the application can not only meet the large-scale culture of stem cells, but also be used for the industrial production of in vitro diagnostic reagent raw materials such as recombinant antigen, viral vector, recombinant protein and monoclonal antibody, with wide cell adaptability, low production cost, significant economy, stable process, and strict requirements of FDA / EMA for biological products.

Provided is a method for large-scale preparation of a purified preparation of a recombinant lentiviral vector at the GMP grade. The method comprises: (a) providing raw material feed liquid to be purified that comprises recombinant viral vectors; (b) carrying out a microfiltration treatment on the feed liquid to obtain a microfiltered filtrate comprising the recombinant viral vectors; (c) optionally concentrating the filtrate to obtain a concentrated filtrate; (d) purifying the filtrate obtained in the previous step by means of chromatography to obtain a crude pure product comprising the recombinant viral vectors; and (e) subjecting the crude pure product obtained in the previous step to liquid exchange and elaborate purification to obtain the purified recombinant viral vectors.

The present invention provides an adeno-associated virus (AAV) vector gene therapy for use in treating a monogenic form of nephrotic syndrome, wherein the AAV vector comprises a NS-associated transgene and minimal nephrin promoter NPHS1 or podocin promoter NPHS2.

The application discloses an application of a nuclear receptor target gene in preparation of a biological preparation for blocking activation of diapause larvae, and relates to the field of biotechnology and parasite prevention and control. According to two nuclear receptor targets of Haemonchus contortus HCON_00101910 (regulating activity of worms) and HCON_00023750 (regulating development of larvae), the two nuclear receptor targets are combined with host progesterone and estradiol and are activated, constitute a key signal path in the spring activation process of diapause larvae, and have no homologous genes in mammals, and the safety is excellent. By constructing specific shRNA lentivirus vectors (the silencing efficiency is all greater than or equal to 60%) targeting the two genes, single target or double target synergistic intervention is realized, and the activity-development double key links of worms in the host body can be specifically blocked. The application provides a new paradigm of preventive prevention and control with high specificity and green safety, and provides core technical support for solving the seasonal epidemic problem of blood fluke disease.

The application discloses a CAR-NK cell based on a dendritic molecule and a preparation method and application thereof. The CAR-NK cell is an assembly formed by the dendritic molecule, effectively loads nucleic acid expressing CAR through electrostatic interaction, forms a stable and uniform delivery system with nanometer size, is safely and effectively delivered to NK cells by virtue of the dendritic molecule, and prepares the CAR-NK cell with high-efficiency target gene expression, so as to solve the problems of low NK cell transduction efficiency and safety of a virus vector and improve the curative effect of the CAR-NK cell. The use of the dendritic molecule for transfection does not affect the phenotype of the NK cell and surface activation and inhibition receptors thereof. The CAR-NK cell prepared based on the dendritic molecule has a remarkable target cell killing efficiency in vitro and a remarkable anti-tumor (Raji-Luc transplanted tumor model) effect in vivo.

The present application provides a kind of PRMT5 inhibitor, PRMT5 inhibitor includes: recombinant adeno-associated virus vector AAV-shPrmt5 and GSK3326595;Recombinant adeno-associated virus vector AAV-shPrmt5 includes the shRNA of targeting PRMT5, the nucleotide sequence of shRNA is as shown in SEQ ID No:1.The present application also provides the application of the above-mentioned PRMT5 inhibitor in the preparation of drug for improving primary biliary cholangitis, PRMT5 inhibitor is all PRMT5 gene / protein as target point, by inhibiting the expression of PRMT5 gene / protein, reduce liver tissue and spleen tissue Tfh cell and GC B cell, serum autoantibody and transaminase level, can effectively delay the disease progression of primary biliary cholangitis.

This invention discloses the application of a PDLIM2 gene overexpression viral vector in the preparation of drugs for treating podocyte disease, wherein the nucleotide sequence of the PDLIM2 gene is shown in SEQ ID NO:1. This invention is the first to discover and verify the core role of the PDLIM2 gene in podocyte protection, confirming its significant downregulation in DKD and FSGS disease models, and demonstrating through functional experiments that PDLIM2 overexpression can effectively stabilize the podocyte cytoskeleton, inhibit apoptosis, and reduce proteinuria, thus establishing it as a key target gene for the treatment of podocyte disease. This invention is the first to intervene at the gene level in the core link of podocyte damage—cytoskeleton stability. By specifically upregulating the expression of PDLIM2 in podocytes, it directly enhances their intrinsic cytoskeleton support and anti-damage ability, achieving a fundamental shift in treatment strategy from "symptomatic support" to "causal repair."