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75results about How to "Efficient transduction" patented technology

Improved rAAv vectors

Disclosed are methods for the use of therapeutic polypeptide-encoding polynucleotides in the creation of transformed host cells and transgenic animals. In particular, the use of recombinant adeno-associated viral (rAAV) vector compositions that specifically target mammalian cells, such as pancreatic islets cells, that express low-density lipoprotein receptors on their cell surface. The disclosed vectors comprise one or more polynucleotide sequences that express one or more mammalian polypeptides having therapeutic efficacy in the amelioration, treatment and / or prevention of AAT- or cytokine polypeptide deficiencies, such as for example in diabetes and related diseases, as well as a variety of autoimmune disorders including, for example, lupus and rheumatoid arthritis.
Owner:UNIV OF FLORIDA RES FOUNDATION INC

AAV vectors for gene delivery to the lung

Methods of making and using recombinant AAV vectors and virions for gene delivery to the lung are described. The recombinant AAV virions are derived from caprine AAV and bovine AAV, both of which display tropism for lung tissue.
Owner:AVIGEN

Methods and compositions relating to improved lentiviral vector production systems

ActiveUS7629153B2Increasing its biosafetySafe transfection and transductionVectorsGenetic material ingredientsTranscriptional Regulatory ElementsTransgene
The present invention provides HIV-derived lentivectors which are multiply modified to create highly safe, efficient, and potent vectors for expressing transgenes for gene therapy. The lentiviral vectors comprise various combinations of an inactive central polypurine tract, a stuffer sequence, which may encode drug susceptibility genes, and a mutated hairpin in the 5′ leader sequence that substantially abolishes replication. These elements are provided in conjunction with other features of lentiviral vectors, such as a self-inactivating configuration for biosafety and promoters such as the EF1α promoter as one example. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element. These vectors therefore provide useful tools for genetic treatments for inherited and acquired disorders, gene-therapies for cancers and other disease, the creation of industrial and experimental production systems utilizing transformed cells, as well as for the study of basic cellular and genetic processes.
Owner:RES DEVMENT FOUND

Photochemical activation of surfaces for attaching biomaterial

A water-soluble photo-activatable polymer including: a photo-activatable group adapted to be activated by an irradiation source and to form a covalent bond between the water-soluble photo-activatable polymer and a matrix having at least one carbon; a reactive group adapted to covalently react with a biomaterial for subsequent delivery of the biomaterial to a cell; a hydrophilic group; and a polymer precursor. A composition including a monomolecular layer of the water-soluble photo-activatable polymer and a matrix having at least one carbon, wherein the monomolecular layer is covalently attached to the matrix by a covalent bond between the photo-activatable group and the at least one carbon. The composition further includes a biomaterial having a plurality of active groups, wherein the biomaterial is covalently attached to the monomolecular layer by covalent bonding between the active groups and reactive groups. Also provided is a method for delivery of a biomaterial to a cell.
Owner:THE CHILDRENS HOSPITAL OF PHILADELPHIA

Gene delivery system and methods of use

InactiveUS20050002903A1Reduce and eliminate native pathogenic potentialEfficient transductionBiocideVectorsHeterologousGene delivery
A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.
Owner:UNIV OF SOUTHERN CALIFORNIA

Serotype of adenovirus and uses thereof

InactiveUS20050232900A1Avoid and diminish immune responseImmune responseBiocideGenetic material ingredientsCell bindingSerotype
Adenovirus serotypes differ in their natural tropism. The adenovirus serotypes 2, 4, 5 and 7 all have a natural affiliation towards lung epithelia and other respiratory tissues. In contrast, serotypes 40 and 41 have a natural affiliation towards the gastrointestinal tract. The serotypes described differ in at least capsid proteins (penton-base, hexon), proteins responsible for cell binding (fiber protein), and proteins involved in adenovirus replication. This difference in tropism and capsid protein among serotypes has led to the many research efforts aimed at redirecting the adenovirus tropism by modification of the capsid proteins.
Owner:JANSSEN VACCINES & PREVENTION BV

AAV virions with decreased immunoreactivity and uses therefor

ActiveUS20080008690A1Decreased immunoreactivityEfficient transductionVirusesHydrolasesVirosomeSerotype
Methods of making and using recombinant AAV virions with decreased immunoreactivity are described. The recombinant AAV virions include mutated capsid proteins or are derived from non-primate mammalian AAV serotypes and isolates that display decreased immunoreactivity relative to AAV-2.
Owner:GENZYME CORP

Universal donor chimeric antigen receptor cells

Disclosed are allogeneic cells useful for the treatment of cancer in a universal donor, off the shelf, manner. In one embodiment of the invention cord blood derived T cell progenitors are matured with anti-CD3 and anti-CD28, interleukin-7 and transfected with a construct encoding a chimeric antigen receptor (CAR) targeting a tumor antigen or a tumor endothelial associated antigen on the antigen binding domain. The intracellular domain containing CD3 zeta chain and at least one shRNA domain encoding a transcript which generates at least one siRNA capable of inhibiting expression of HLA I and / or HLA II. In another embodiment mesenchymal stem cells are transfected with CAR to enhance migration into tumors and induce tumor death, reduction of inflammation, or immune sensitization. In another embodiment universal donor CAR-MSC are disclosed.
Owner:BATU BIOLOGICS

Hyperbranched polyamino acid, preparation method and application thereof

The invention relates to hyperbranched polyamino acid. The chemical composition is as follows: in the formula of (Lys-)a-(-Arg-)b-(-His-)c-(-X)d, Lys, Arg and His are respectively lysine, arginine and histidine, X is one of or a plurality of any amino acids except for the amino acids above, wherein a is not less than 0.2 and not more than 0.95, b is not less than 0 and not more than 0.8, c is not less than 0 and not more than 0.8, d is not less than 0 and not more than 0.3, and the sum of a, b, c and d is 1. The preparation method is as follows: uniformly mixing lysine or lysine salt with other amino acids or the amino acid salts contained in the hyperbranched polyamino acid to react for 2-64h at the temperature of 130-180 DEG C and then obtain yellow solid, and finally purifying the coarse product with a precipitation method to obtain the hyperbranched polyamino acid. The hyperbranched polyamino acid obtained in the invention can be used as nonviral gene carrier for the transduction of huamn or animal cell gene, and the hyperbranched polyamino acid nonviral gene carrier provided by the invention is reasonable in design, simple in preparation method, high in transfection efficiency, low cytotoxicity and favorable transduction efficiency in the presence of blood serum.
Owner:WUHAN UNIV

Lentivirus based vector and vector system

The present invention relates to retroviral vectors which will infect and confer efficient gene transfer to non-dividing cells including the cells of the central nervous system. The vector system of the present invention is useful as a gene transfer vehicle for gene therapy, i.e. of the central nervous system.
Owner:BAVARIAN NORDIC RES INST AS

Photochemical activation of surfaces for attaching biomaterial

A water-soluble photo-activatable polymer including: a photo-activatable group adapted to be activated by an irradiation source and to form a covalent bond between the water-soluble photo-activatable polymer and a matrix having at least one carbon; a reactive group adapted to covalently react with a biomaterial for subsequent delivery of the biomaterial to a cell; a hydrophilic group; and a polymer precursor. A composition including a monomolecular layer of the water-soluble photo-activatable polymer and a matrix having at least one carbon, wherein the monomolecular layer is covalently attached to the matrix by a covalent bond between the photo-activatable group and the at least one carbon. The composition further includes a biomaterial having a plurality of active groups, wherein the biomaterial is covalently attached to the monomolecular layer by covalent bonding between the active groups and reactive groups. Also provided is a method for delivery of a biomaterial to a cell.
Owner:THE CHILDRENS HOSPITAL OF PHILADELPHIA

Serotype of adenovirus and uses thereof

The invention provides a gene delivery vehicle and a gene of interest comprising at least one Ad35 element or a functional equivalent thereof, responsible for avoiding or diminishing neutralizing activity against adenoviral elements by the host to which the gene is to be delivered. A functional equivalent / homologue of an Ad35 (element) includes an adenovirus (element) which, like adenovirus 35, encounters pre-existing immunity in less than about 10% of the hosts to which it is administered for the first time, or which is capable in more than about 90% of the hosts to which it is administered of avoiding or diminishing the immune response.
Owner:JANSSEN VACCINES & PREVENTION BV

Pseudotyping of retroviral vectors, methods for production and use thereof for targeted gene transfer and high throughput screening

ActiveUS20100189690A1Increase in titer and transduction efficiencyHigh transduction efficiencySsRNA viruses negative-senseBiocideSurface markerMorbillivirus
The invention relates to the pseudotyping of retroviral vectors with heterologous envelope proteins derived from the Paramyxoviridae family, genus Morbillivirus, and various uses of the resulting vector particles. The present invention is based on the unexpected and surprising finding that the incorporation of morbillivirus F and H proteins having truncated cytoplasmic tails into lentiviral vector particles, and the complex interaction of these two proteins during cellular fusion, allows for a superior and more effective transduction of cells. Moreover, these pseudotyped vector particles allow the targeted gene transfer into a given cell type of interest by modifying a mutated and truncated H protein with a single-chain antibody or ligand directed against a cell surface marker of the target cell.
Owner:BUNDESREPUBLIK DEUTLAND LETZTVERTRETEN DURCH DEN PRASIDENTEN DES PAUL EHRLICH INSTITUTS

Multi-band antenna apparatus disposed on a three-dimensional substrate, and associated methodology, for a radio device

ActiveUS20090051615A1Efficient transductionStable and relatively wide resonant frequency band characteristic and radiation patternSimultaneous aerial operationsAntenna supports/mountingsMulti bandDielectric
Antenna apparatus, and an associated methodology, for a multi-frequency-band-capable radio device, such as a quad-band mobile station. The antenna apparatus is formed from a three-dimensional rectilinear non-conductive dielectric antenna substrate, such as cube. An elongated radiation element is disposed over multiple surfaces of the antenna substrate. A T-shaped impedance matching element located at the end of the radiation element permits the antenna input impedance to be matched to a communications device. The length of the radiation element is selected to be substantially equal to a quarter wavelength of the lowest frequency band at which the antenna operates.
Owner:MALIKIE INNOVATIONS LTD

Adenovirus vector containing a heterologous peptide epitope in the hi loop of the fiber knob

The present invention provides means to modify the tropism of recombinant adenoviral vectors using genetic methods to alter the adenoviral fiber cell-binding protein. The present invention generates an adenovirus with modified fiber gene such that novel tropism is achieved. This recombinant adenovirus has a fiber gene modified in the HI loop domain.
Owner:UAB RES FOUND

Methods and compositions relating to improved lentiviral vector production systems

ActiveUS20100062524A1Increasing its biosafetySafe transfection and transductionVectorsGenetic material ingredientsTranscriptional Regulatory ElementsTransgene
The present invention provides HIV-derived lentivectors which are multiply modified to create highly safe, efficient, and potent vectors for expressing transgenes for gene therapy. The lentiviral vectors comprise various combinations of an inactive central polypurine tract, a stuffer sequence, which may encode drug susceptibility genes, and a mutated hairpin in the 5′ leader sequence that substantially abolishes replication. These elements are provided in conjunction with other features of lentiviral vectors, such as a self-inactivating configuration for biosaftey and promoters such as the EF1α promoter as one example. Additional promoters are also described. The vectors can also comprise additional transcription enhancing elements such as the wood chuck hepatitis virus post-transcriptional regulatory element. These vectors therefore provide useful tools for genetic treatments for inherited and acquired disorders, gene-therapies for cancers and other disease, the creation of industrial and experimental production systems utilizing transformed cells, as well as for the study of basic cellular and genetic processes.
Owner:RES DEVMENT FOUND

Gene delivery system and method of use

InactiveUS20120052554A1Reduce and eliminate native pathogenic potentialEfficient transductionVectorsFermentationGene deliveryReplication Competent Retrovirus
A recombinant replication competent retrovirus for gene deliver and gene therapy is provided. The recombinant retrovirus has a heterologous nucleic acid sequence, a sequence encoding a cell- or tissue-specific ligand or a sequence for transcriptional targeting, or a combination of both a cell- or tissue-specific ligand and a cell- or tissue-specific transcriptional targeting sequence.
Owner:UNIV OF SOUTHERN CALIFORNIA

Preparation method and application of capsid-protein-mutant double-stranded recombinant adeno-associated virus containing mediated-membrane-stabile CD40L gene

The invention discloses a nucleotide sequence and an amino acid sequence of a human derived membrane-stabilization mutant gene CD40L-M, and a plasmid carrier pdsAAV-CB-CD40L-M. The invention further discloses a recombinant adeno-associated virus scAAV2 / 5-Y719F-CD40L-M using CD40L-M as a target gene and highly-efficiently transducing lung cancer cells, a preparation method thereof, and an application thereof in preparing anti-cancer drugs. In vitro and in vivo tests of transduction of the CD40L-M gene into the lung cancer cells show that generation of the sCD40L is substantially reduced, a traditional AAV transduction efficiency is improved by using the recombinant adeno-associated virus scAAV2 / 5-Y719F-CD40L-M, the lung cancer cells can be highly-efficiently transduced, and the CD40L-M gene induces retardance of cell cycles, promotes cell apoptosis, induces immunization activation, inhibits growth of lung cancer in the body and reduces side effects such as liver and kidney damage.
Owner:JIANGSU PROVINCE HOSPITAL

Fiber-modified adenoviral vectors for enhanced transduction of tumor cells

Adenoviral vectors which effectively transduce primary tumor cells are provided. The adenoviral vectors comprise a chimeric adenovirus fiber protein which includes at least a portion of a Subgroup C adenovirus fiber shaft and at least a portion of a Subgroup B adenovirus or serotype 37 adenovirus head, wherein the head region binds CD46.
Owner:细胞基因系统有限公司

Gene transfer methods

Improved methods for transferring a gene into target cells by using a retrovirus, wherein the gene transfer efficiency is improved and the target cells are efficiently transformed by binding the retrovirus to a functional substance which is immobilized on as carrier and having an activity of binding to retroviruses followed by washing; using an antibody capable of specifically recognizing cells, laminin or mannose-rich type sugar chain as a substance having an activity of binding to the target cells; pre-treating the target cells so as to inactivate transferrin receptor, or introducing a new functional group into the functional substance.
Owner:TAKARA HOLDINGS
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