2681results about "SsDNA viruses" patented technology

The invention discloses chicken infectious anemia virus-like particles as well as a preparation method and application thereof. The invention discloses a virus-like particle vaccine for preventing chicken infectious anemia. The virus-like particle vaccine comprises VP1 and VP2 proteins of chicken infectious anemia viruses. Chicken infectious anemia VP1 and VP2 proteins are expressed by using a baculovirus expression system, immunoblotting shows that the two proteins are successfully expressed in sf9 cells, electron microscope observation finds that the expressed proteins can be autonomously assembled into complete chicken infectious anemia virus-like particles, and the chicken infectious anemia virus-like particles have a space structure similar to that of an original virus and can be used for preparing chicken infectious anemia virus-like particles. Meanwhile, the virus-like particles have the advantages of high titer, high safety, capability of stimulating humoral immunity and cellular immunity and the like. The preparation method disclosed by the invention is simple, can be used for preparing the antigen protein of the chicken infectious anemia virus on a large scale, is high in expression quantity and short in time consumption, greatly reduces the production cost, and is suitable for large-scale production.

The present disclosure provides codon optimized GBA1 polynucleotides encoding a GCase protein, wherein a portion of the coding sequence deviates from the wild type. The disclosure also provides expression constructs, vectors, viral particles or compositions containing the disclosed polynucleotides. In addition, methods and uses of these polynucleotides, expression constructs, vectors, viral particles or compositions are also provided, including the treatment of diseases or conditions associated with GCase deficiency.

Described herein are muscle-specific targeting moieties and compositions including the muscle specific targeting motifs. Also described herein are uses of the muscle-specific targeting motifs and compositions including the muscle specific targeting moieties. In some embodiments, the muscle-specific targeting moieties and compositions including the muscle specific targeting moieties can be used to direct delivery of a cargo to a muscle cell.

The present disclosure relates to novel adeno-associated adenovirus (AAV) vectors comprising targeting peptides. More particularly, the present disclosure relates to an adeno-associated serotype 2 virus vector, AAV2, comprising a transgene encoding LIGHT wherein the viral capsid of the AAV2 vector comprises a targeting peptide that alters its tropism to target tumor endothelial cells; and a use of the carrier. The disclosure also relates to the use of said vectors in therapy, in particular in the treatment of cancer.

Among other things, provided herein are systems that replace the natural random integration activity of a retrovirus with site-specific integration machinery. This approach allows for a more precise targeting of a gene of interest into a human genome, e.g., for therapeutic purposes. The system may include integration-deficient retrovirus (e.g., lentivirus) (IDLV), in which the natural integration activity has been reduced (e.g., by mutation to the viral integrase polypeptide). Instead, the system may comprise a site-specific recombinase (e.g., a serine recombinase, e.g., a serine integrase) capable of directing insertion of a template DNA, or portion thereof, into a desired site in the human genome.

Described herein are targeting moieties that can be capable of specifically targeting muscle cells and can include an n-mer motif. In some embodiments, the n-mer motif contains an RGD motif. Also described herein are vector systems, particles, polypeptides that can encode and / or contain one or more targeting moieties. Also described herein are methods of delivering a cargo to a cell, such as a muscle cell, using one or more of the targeting moieties described herein.

An engineered producer cell comprising an inactivating mutation in one or more endogenous REP binding sites is provided, as are methods for producing the engineered producer cell and using the engineered producer cell to produce a recombinant viral vector and reduce producer cell genomic DNA contamination of a recombinant adeno-associated virus vector preparation.

Described herein are methods and compositions that utilize a CRISPR / Cas system. In some cases, the utilization includes the use of such systems for gene activation or interference.

Provided herein are novel AAV capsids and rAAV comprising the same. In one embodiment, vectors employing a novel AAV capsid show increased transduction of a selected target tissue as compared to a prior art AAV.

The present disclosure provides evolved and engineered reverse transcriptase variants and Cas9 variants with improved properties (e.g., improved editing efficiency when used in a guided editor context). The disclosure also provides fusion proteins comprising the reverse transcriptase variants and Cas9 variants described herein, including, for example, a guided editor. The disclosure also provides polynucleotides encoding the reverse transcriptase variants, Cas9 variants, and guide editors provided herein, as well as vectors comprising such polynucleotides. The disclosure also provides pharmaceutical compositions and cells comprising the reverse transcriptase variants, Cas9 variants, and a guided editor described herein. The present disclosure also provides methods and uses relating to the reverse transcriptase variants, Cas9 variants, and guide editors described herein.

The invention discloses a recombinant adeno-associated virus vector for targeted delivery of VEGF-C (vascular endothelial growth factor-C) and application of the recombinant adeno-associated virus vector in promoting endocardial differentiation to treat coronary heart disease. The rAAV vector comprises transgene for coding VEGF-C protein or VEGF-C protein functional variant and a tissue-specific promoter operably connected to the transgene, wherein the tissue specific promoter is capable of driving the transgene to be specifically expressed in the heart endothelial cells. According to the treatment method developed by the invention, specific expression of the Vegfc gene in the heart is driven by using an ICAM2 promoter with targeting to endothelial cells, and a remarkable curative effect is shown in a myocardial infarction model mouse. The therapeutic scheme provides a new effective strategy for intervention and treatment of myocardial infarction and chronic heart failure, and has the advantages of high safety, long gene expression duration, extremely low immune response to a host and the like.

The disclosure is directed in part to variant capsid polypeptides that can be used to deliver payloads.

The present invention relates to engineered liver-specific core promoters, synthetic promoters (which contains the engineered core promoters and enhancers), expression vectors (which contains the synthetic promoter), as well as methods of using the promoter or the expression vector thereof to address the need in the field, including treatment of various genetic diseases or conditions associated with the liver. In some embodiments, the liver-specific promoter includes continuous or discontinuous genome sequences from SERPINA1 genome.

The disclosure relates to compositions, methods, and processes for the preparation, use, and / or formulation of adeno-associated virus capsid proteins, wherein the capsid proteins comprise targeting peptide inserts for enhanced tropism to a target tissue.

Provided are techniques for generating expression products using one or more nucleic acid concatemers that include tandem repeats of a nucleic acid sequence encoding the expression product or products. In one embodiment, different expression products may be co-expressed using a concatemer mixture of a first nucleic acid concatemer and a second nucleic acid concatemer having a predefined ratio to one another.

Provided herein are modified nucleic acids comprising modified MTM1 genes, modified muscle-specific promoters, e.g., MHCK7, or a combination thereof. Also provided herein are expression cassettes, AAV vector genomes, and AAV particles comprising the same. Further provided herein are methods of delivering MTM1 to a muscle cell and methods of treating XLMTM.

Waning immunity induced by first-generation Spike-alone-based COVID-19 has failed to prevent immune escape by many variants of concern (VOCs) that emerged from 2020 to 2024, resulting in a prolonged COVID-19 pandemic. Thus, a next-generation Coronavirus (CoV) vaccine incorporating highly conserved non-Spike SARS-CoV-2 antigens is described herein. Conserved non-Spike T cell antigens in combination with a Spike antigen encapsulated in lipid nanoparticles: (i) Induced high frequencies of lung-resident antigen-specific CXCR5+CD4+ T follicular helper cells, GzmB+CD4+ and GzmB+CD8+ cytotoxic T cells, and CD69+IFN-γ+TNFα+CD4+ and CD69+IFN-γ+TNFα+CD8+ effector T cells; and (ii) Reduced viral load and COVID-19-like symptoms caused by various VOCs. The combined antigen / LNP-based pan-CoV vaccine could be rapidly adapted for clinical use to confer broader cross-protective immunity against emerging highly mutated and pathogenic VOCs.

Aspects of the disclosure relate to recombinant gene editing complexes comprising a recombinant gene editing protein and guide RNA (gRNA) that specifically hybridizes to a region of a C90RF72 gene (e.g., a region flanking a G4C2 repeat or within a exonic region of the gene).

This document relates to materials and methods for controlling ligand gated ion channel (LGIC) activity. For example, modified LGICs including at least one LGIC subunit having a modified ligand binding domain (LBD) and / or a modified ion pore domain (IPD) are provided. Also provided are exogenous LGIC ligands that can bind to and activate the modified LGIC, as well as methods of modulating ion transport across the membrane of a cell of a mammal, methods of modulating the excitability of a cell in a mammal, and methods of treating a mammal having a channelopathy.

According to various aspects of this disclosure, the present disclosure relates to methods for reducing kidney inflammation, reducing kidney fibrosis, reducing oxidative stress in the kidney, preventing or reducing the likelihood of chronic kidney disease (CKD), treating or preventing acute kidney injury (AKI) or treating a kidney disease in a subject in need thereof comprising administering, e.g., intramuscularly, to the subject a recombinant adeno-associated virus (rAAV) vector, wherein the rAAV vector comprises a vector genome comprising a nucleotide sequence encoding a Fibroblast growth factor 21 (FGF21) or a functional fragment thereof and an AAV capsid (e.g., AAV1 serotype), optionally, wherein the subject does not suffer from diabetes.

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 NPHIS1 or podocin promoter NPHIS2.

Provided herein is a system for delivering genetical material, preferably subcutaneously, and its various elements. Also provided are related compositions and methods.

The present disclosure provides preventing and / or treating hearing loss in an individual in need thereof comprising administering a composition comprising one or more viral vectors comprising a nucleic acid encoding COL18A1 protein, SPARC protein, and IGFBP2 protein. The present disclosure also provides method for preventing and / or treating hearing loss in an individual in need thereof comprising administering an effective amount of COL18A1, SPARC, and IGFBP2 proteins to the inner ear of the individual.

The invention relates to the technical field of biology, in particular to an MDCK cell strain and application of the MDCK cell strain in preparation of influenza vaccines. The invention provides an MDCK cell strain, the preservation number of which is CCTCC NO: C202504. The cell strain not only can maintain high sensitivity and high yield to influenza viruses and ensure the yield and quality of vaccines, but also can remarkably improve the biological safety of cell matrix influenza vaccines due to elimination of tumorigenicity risk, and has a good application prospect. And a key technical support is provided for promoting domestic research and development and marketing of influenza vaccines based on cell culture.

This present disclosure provides adeno-associated viral vectors, recombinant adeno-associated virus (rAAV), and methods of their use in gene therapy for treating propionic acidemia (PA). Also provided are pharmaceutical compositions comprising a recombinant adeno-associated virus of the invention and a pharmaceutically acceptable carrier or excipient. These pharmaceutical compositions may be useful in gene therapy for the treatment of PA caused by mutations in propionyl-CoA carboxylase α-subunit (PCCA) or mutations in propionyl-CoA carboxylase β-subunit (PCCB).

The invention relates to haptoglobin or a nucleic acid encoding haptoglobin for use in treating or preventing of exaggerated erectile response and / or preventing permanent erectile dysfunction. In addition, the invention relates a pharmaceutical composition for use in treating or preventing exaggerated erectile response and / or preventing permanent erectile dysfunction, wherein the pharmaceutical composition comprises an adeno-associated viral (AAV) vector with a transgene encoding a haptoglobin gene. The exaggerated erectile response may be priapism, for example priapism associated with sickle cell disease (SCD).

Methods are disclosed for producing high-titer recombinant adeno-associated virus (rAAV) using a modified adenovirus-based helper nucleic acid. One aspect provided herein is a human adenovirus 5 (hAd)-based nucleic acid that does not contain one or more of (a) an E4 region having E4-ORF6 / 7, (b) a viral associated (VA) RNA region, (c) an E2A region having L4-22K and L4-33K, (d) at least one packaging protein, (e) at least one structural protein, (f) a major late promoter (MLP), (g) an E1 region, and / or (h) an E3 region.

The invention relates to gene therapy of ocular conditions. Described herein are compositions and methods for delivering therapeutic products, such as therapeutic proteins (e.g., antibodies), therapeutic RNAs (e.g., shRNAs, siRNAs, and miRNAs), and therapeutic aptamers, to the retinal / vitreous humor of the eye of a human subject to treat ocular conditions, involving, for example, recombinant viral vectors, such as recombinant adeno-associated virus (rAAV) vectors.

The disclosure relates to compositions and methods for altering, e.g., enhancing, the level of frataxin protein via delivery using an adeno-associated viral (AAV) capsid variant. The compositions and methods of the present disclosure are useful in the treatment of subjects who have, have been diagnosed with having, or are at risk of having a disorder associated with frataxin (FXN) deficiency, e.g., Friedreich's Ataxia.

A polynucleotide comprising a nucleotide sequence encoding an engineered sacsin, wherein the engineered sacsin comprises a UbL domain or variant thereof, an SRR domain or variant thereof, a DnaJ domain or variant thereof and an HERN domain or variant thereof, and wherein the nucleotide sequence encoding the engineered sacsin is less than or equal to about 4000 bp in length.