59 results about "Viral procapsid" patented technology

The technology described herein provides variant adeno-associated viral capsid polypeptides and viruses comprising the same. Further provided herein are methods for delivering a viral payload using viruses comprising variant capsid polypeptides described herein. Described herein are viral vectors comprising a variant sequence of the capsid gene, VP1. In particular, viral vectors with capsid polypeptide mutations that modify tropism of the viral particles relative to particles with wild-type capsid polypeptide are described.

The application discloses an integrin-reformed specific heart and muscle targeting AAV9 dominant mutant. The application provides an adeno-associated virus capsid protein, which comprises a targeting peptide containing an amino acid sequence as shown in any one of SEQ ID NO: 1-28. According to the RGD sequence binding with integrin, the polypeptide specific binding principle, the random amino acid carrying and the construction of the diversified AAV capsid library, and the high-throughput screening of the AAV with the suitable capsid structure, the application successfully obtains a new adeno-associated virus vector with high heart and / or muscle transduction efficiency, and the new adeno-associated virus vector exhibits better heart and / or skeletal muscle targeting and transduction efficiency in an animal model, and the off-target expression in non-target tissues is significantly reduced, and the new adeno-associated virus vector has important scientific value and commercial prospect.

Recombinant adeno-associated viral (AAV) capsid proteins are provided. Methods for generating the recombinant adeno-associated viral capsid proteins and a library from which the capsids are selected are also provided.

Provided herein are methods and systems for detecting polynucleotide sequences encoding viral capsids in metagenomic data. The methods and systems disclosed herein may include a sequence alignment-based module, a gene-based data processing module, and further characterization of putative viral sequences to identify novel polynucleotide sequences encoding viral capsids in metagenomic data.

The present application relates to (i) a variant adeno-associated virus (AAV) capsid polypeptide comprising a peptide insertion in the variable region IV or in the variable region VIII relative to a wild-type AAV capsid polypeptide, wherein the peptide insertion comprises an amino acid sequence selected from the group consisting of SEQ ID NOs:1-29 or an amino acid sequence having at least 70% sequence identity thereto, (ii) an isolated nucleic acid encoding the aforementioned variant polypeptide, (iii) a recombinant polynucleotide comprising the aforementioned nucleic acid, and (iv) an isolated cell comprising the aforementioned polypeptide, nucleic acid or recombinant polynucleotide. The present application further relates to (v) an adeno-associated virus (AAV) vector comprising the aforementioned variant polypeptide, (vi) a pharmaceutical composition comprising the aforementioned AAV vector as well as (vii) the use of the aforementioned vector or pharmaceutical composition in preventing or treating an ocular disease. Finally, the present application relates to (viii) a method of delivering a heterologous nucleic acid to a retinal cell and (ix) a method of delivering a heterologous nucleic acid to the eye of a subject.

The invention relates to a helper adenovirus genome wherein expression of one or more of the adenoviral early E1 genes, E2 genes including DNA-binding protein (DBP), pre-terminal protein (pTP) and DNA polymerase, or production of functional proteins are regulated. The invention also relates to a process for delivery of the helper-dependent or gutless adenovirus vector genome and modulating replication of the helper virus DNA in the manufacturing cells, in which to allow a sufficient length of time before excision, cutting, or recombination to removal of the viral packaging signal DNA sequence from the helper adenovirus genome prior to induction of viral DNA replication, and expression of helper proteins that are required for adenovirus capsid particle formation and packaging, or encapsidation, of the helper-dependent or gutless adenovirus vector genomes, while preventing the packaging, or encapsidation, of the helper adenoviral genome into preform adenoviral capsids.

The present disclosure relates to compositions of surface modified viral capsids having high physical titers. The present disclosure also relates to a method of making a surface modified viral capsid using an efficient process of (a) reacting (i) a viral capsid comprising a plurality of surface accessible primary amines and (ii) a capsid-reactive linker comprising a terminal tetrafluorophenyl (TFP) ester and a first member of a cross-linking agent reactive pair, to provide a composition comprising a surface functionalized viral capsid; and (b) reacting the composition comprising the surface functionalized viral capsid with a functionalized ligand comprising a second member of the crosslinker reactive pair.

The present invention relates to the technical field of prevention and control of aquatic animal diseases, and specifically relates to a nanocomposite for a nervous necrosis virus, a preparation method therefor, and the use thereof. Provided in the present invention is a nanocomposite consisting of poly(lactic-co-glycolic acid), polyethyleneimine and a siRNA. Also provided in the present invention are a preparation method for the nanocomposite and the use thereof in the preparation of a drug for improving the survival rate of fish eggs infected with a nervous necrosis virus. The present invention constructs a vector for a capsid protein of the nervous necrosis virus by means of simulating the nervous necrosis virus and expresses same in cells, and screens out a siRNA that can effectively inhibit the expression of the viral capsid protein, thus constructing the nanocomposite for the nervous necrosis virus. The nanocomposite can block NNVs at the embryonic phase (fertilized eggs), thereby improving the survival rate of fish fry, and further opening up new possibilities for the treatment of early stage diseases in fish fry. The method is innovative in the field of artificial breeding of aquatic animals and provides new ideas for virus prevention and control in the field of aquaculture.

This application provides a novel differentiation factor for direct cross-differentiation from somatic cells to motor neurons, a composition containing the differentiation factor, a method for direct cross-differentiation using the composition, and uses for the novel differentiation factor. [Solution] A composition for direct cross-differentiation from somatic cells to induced motor neurons, the composition comprising (A) the Gsta4 protein or nucleic acid coding therefor; and (B) one or more proteins selected from Ascl1, Brn2, Myt1L, Hb9, ISL1, Lhx3, Ngn2 and NeuroD1 or nucleic acid coding therefor; and further comprising a viral capsid for transmitting (A) or (B).

The invention relates to a helper adenovirus genome wherein expression of one or more of the adenoviral early E1 genes, E2 genes including DNA-binding protein (DBP), pre-terminal protein (pTP) and DNA polymerase, or production of functional proteins are regulated. The invention also relates to a process for delivery of the helper-dependent or gutless adenovirus vector genome and modulating replication of the helper virus DNA in the manufacturing cells, in which to allow a sufficient length of time before excision, cutting, or recombination to removal of the viral packaging signal DNA sequence from the helper adenovirus genome prior to induction of viral DNA replication, and expression of helper proteins that are required for adenovirus capsid particle formation and packaging, or encapsidation, of the helper-dependent or gutless adenovirus vector genomes, while preventing the packaging, or encapsidation, of the helper adenoviral genome into preform adenoviral capsids.

The present invention relates to the fields of medicine, molecular biology, and gene therapy. In particular, the invention relates to novel recombinant adeno-associated virus capsids, uses thereof and methods of manufacturing.

Provided herein are compositions and methods of utilizing engineered viral capsid for therapeutic delivery. Also provided herein are methods of using the provided compositions and methods for delivering therapeutic for treating diseases.

Provided herein are targeting peptides and vectors containing a sequence that encodes the targeting peptides that deliver agents to specific substructures in the brain. Specifically, the targeting peptide is a component of a modified, sequence-specified adeno-associated virus (AAV) capsid protein further wherein the brain substructure may be the globus pallidus, putamen, internal capsule, caudate, claustrum, substantial nigra, motor cortex, insula,.temporal cortex, thalamus, hippocampus, subiculum, and deep cerebellar nuclei.

A method of estimating a proportion of particles filled with material. The method includes: obtaining an image depicting a plurality of particles; generating a plurality of classes based on the plurality of particles; identifying at least one of the plurality of classes having a central portion with a greater density than a central portion of at least one other of the classes; and estimating a proportion of the plurality of particles that are filled with material based on identifying at least one of the classes having a central portion with high density.

The present invention relates to the field of modular platforms, such as viral vectors. In particular, the present invention relates to a recombinant adeno- associated virus (AAV) capsid or particle comprising an engineered virion protein (VP), wherein the engineered VP comprises at least one cell-type specific binding moiety located in variable region 8 (VR8). The invention also relates to a recombinant AAV particle for use as a medicament.

This invention relates to novel surface modified viral capsids and recombinant virions comprising the same. Furthermore, this invention concerns intermediates for the preparation of surface modified viral capsids. The surface modified viral capsids are designed to selectively and / or more efficiently deliver gene therapy. The surface modified viral capsids, when incorporated into a recombinant virion, can be used to treat an illness that is characterized by genetic abnormality.

The present disclosure generally relates to engineered adeno-associated virus and their uses in T cell engineering. Also disclosed are engineered viral capsid polypeptides, vectors, cells, and kits, related to the engineered adeno-associated virus, and methods related to the uses thereof.

An adeno-associated virus (AAV) capsid protein variant with improved infectivity to target cells and transduction efficiency, and its use are disclosed. Specifically, an AAV2 variant capsid protein selected through directed evolution for gene delivery to retinal cells, a recombinant AAV vector containing the same, and its use as a gene delivery vehicle to retinal cells are disclosed.

The present invention relates to recombinant adeno-associated viruses (rAAVs) having capsid proteins engineered to include amino acid sequences that confer and / or enhance desired properties. In particular, the invention provides engineered capsid proteins comprising peptide insertions from heterologous proteins inserted within or near variable region IV (VR-IV) of the virus capsid, such that the insertion is surface exposed on the AAV particle. The invention also provides capsid proteins that direct rAAVs to target tissues, in particular, capsid proteins comprising peptides that are inserted into surface-exposed variable regions using a method for replacing a stop codon in the capsid gene with such peptide insert. The invention provides such methods for making the rAAV vectors into vector libraries and selecting the rAAV vectors having peptide inserts that target the rAAV to particular tissues of interest, and thus deliver therapeutics for treating disorders.

Some embodiments of the present disclosure are directed to methods and compositions for the treatment of tumors, using a combination of immunotherapeutic agents and tumor-targeting viral capsid protein assemblages comprising anti-cancer molecules conjugated to viral capsid proteins.

Provided are a modified adeno-associated virus capsid protein and the use thereof. The capsid protein comprises a substitution of about 5-8 amino acids as compared with a wild-type AAV capsid protein, and an AAV comprising the mutated capsid protein has increased infectivity to target tissue or target cells (such as heart or cardiomyocytes) than an AAV comprising the unmutated wild-type AAV capsid protein.

Herein is reported a novel variant adeno-associated virus (AAV) capsid protein comprising a heterologous peptide or polypeptide covalently inserted in the GH-loop of the capsid protein relative to a corresponding parental AAV capsid protein, wherein the heterologous peptide or polypeptide comprises a recognition sequence of a transglutaminase and the amino acids residues at positions 452-455 or 453-459 of VP1 of AAV-2 (SEQ ID NO: 65) or at the corresponding positions in the capsid protein of another AAV serotype are replaced by the heterologous peptide or polypeptide or inserted at amino acid position 456.

Virus-Like Particles derived from the subfamilies, Parahepevirinae, which infect trout and salmon, and the Orthohepevirinae, which infect mammals and birds, particularly those of the species Paslahepevirus balayani, which can cause acute hepatitis in humans and several mammalian species, and chronic conditions in immunocompromised patients are also disclosed. Major aspects of the invention relate to compositions of Virus-Like Particles comprising viral capsid proteins capable of assembly in cultured cells that may be purified, disassembled, and reassembled in the presence of other molecules suitable for use as therapeutic drug products to facilitate the targeting and delivery of cargo molecules to specific cells or tissues, or as antigenic agents designed to stimulate responses to heterologous epitopes exposed on the surfaces of Virus-Like Particles. Preferred aspects relate to functional capsids comprising polypeptide sequences comprising one or more amino acid substitutions, insertions, or deletions of amino acid encoded by a consensus of ORF2 genes, wherein said variant polypeptides are functionally-similar or have enhanced properties compared to capsid polypeptides encoded by naturally-occurring viruses obtained from clinical samples or prototype Hepatitis E Viruses (HEV). Other aspects include the design and assembly of modified vectors to facilitate the basic and applied studies leading to the development and commercialization of novel drug products, and as tools advancing the interests of institutions involved in animal and human healthcare.

This invention relates to novel neurotropic adeno-associated virus (AAV) capsid variants. In particular, the invention relates to novel AAV capsid variants that efficiently transduce cells in the central nervous system (CNS) upon systemic administration, while exhibiting reduced transduction to peripheral organs. The invention further relates to a method for identifying AAV capsid variants having one or more desired properties, such as a combination of CNS targeting and peripheral organ detargeting.

Provided is a method of enriching full adenovirus-associated virus (AAV) capsids from a mixture of full AAV capsids and empty AAV capsids, the method comprising providing a sample comprising a mixture of full AAV capsids and empty AAV capsids, subjecting the sample to anion exchange chromatography comprising an elution buffer comprising an equilibration buffer and a salt-containing buffer in an initial ratio that provides an initial conductivity, and changing the ratio of the equilibration buffer and the salt-containing buffer to provide a step gradient conductivity increase of about 0.5-2.0 mS / cm in each step to elute empty AAV capsids and to provide a fluid enriched with full AAV capsids.

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

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

The present invention relates to the fields of medicine, molecular biology, and gene therapy. In particular, the invention relates to novel recombinant adeno-associated virus capsids, uses thereof and methods of manufacturing.

The invention provides a muscle-targeted adeno-associated virus capsid protein, an adeno-associated virus containing the muscle-targeted adeno-associated virus capsid protein, a vector and application. The present disclosure provides an adeno-associated virus capsid protein, which comprises a targeting peptide, and the targeting peptide comprises any one of the following amino acid sequences: SEQ ID NO: 1-19. According to the invention, a reasonable design and directed evolution combined method is adopted, and on the basis of a receptor integrin-RGD polypeptide specific binding principle, random amino acids are carried to adapt to construction and screening of the AAV vector of a capsid structure, so that the problems of poor stability of the capsid structure, change of histocompatibility and the like caused by direct insertion of a sequence are avoided; therefore, efficient transduction of muscular tissues is realized, and a reliable platform is provided for gene therapy.