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48 results about "Adenoassociated virus" patented technology

Method to generate mirrored adenoassociated viral vectors

The present invention describes mirrored adenoassociated virus genomes that can spontaneously fold to form double-stranded DNA structures capable of directing efficient RNA transcription in mammalian cell nuclei. Also described are mirrored adenoassociated viral particles that incorporate the mirrored vector genome and a suitable adenoassociated viral capsid. Further described are DNA templates and methods for producing the mirrored adenoassociated vector genomes and mirrored adenoassociated viral particles. Methods of administering these reagents to mammals are also described as are specific in vitro and in vivo applications where the mirrored adenoassociated virus has unique utility.
Owner:PETRAS OGNJEN

Adenoassociated virus vectors for the treatment of lysosomal storage disorders

The present invention provides new adenoassociated virus vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidoses Type IIIB.
Owner:AUTONOMOUS UNIVERSITY OF BARCELONA +1

Gene therapy medicament for Leber congenital amaurosis

The invention provides a gene therapy medicament for specific expression of retinal pigment epithelium 65 (RPE65) of eyes in order to treat Leber congenital amaurosis. A vector contains a CMV enhancer, a promoter of a human RPE65 gene, a human RPE65 gene coding region and an intron composed sequence, and a poly A sequence and an artificially spliced RPE65 gene enhancer. The recombinant vector is mediated by a single chain and / or double chain adeno-associated virus (AAV), including but not limited to 9 type AAV type 9 (AAV9).
Owner:BEIJING GENECRADLE PHARM CO LTD

Antibody capable of binding to AAV1-13

The invention relates to the field of immunology, in particular to an antibody capable of binding to AV1-13. The antibody can recognize VP1, VP2 and VP3 proteins of all serotypes of AV1-13, is high inaffinity and good in specificity; and the antibody can be more conveniently used for separation and identification of adeno-associated viruses due to the fact that the VP proteins are located on thesurfaces of the adeno-associated viruses.
Owner:OBIO TECH SHANGHAI CORP LTD

Adenoassociated virus vectors for the treatment of mucopolysaccharidoses

The present invention provides new Adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidoses Type II.
Owner:ESTEVE PHARMA SA +1

Adenoassociated virus vectors for the treatment of mucopolysaccharidoses

The present invention provides new adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidoses Type IIID.
Owner:ESTEVE PHARMA SA +1

Avian adenoassociated virus and uses thereof

The present invention provides an Avian adeno-associated virus (AAAV) virus and vectors and particles derived therefrom. In addition, the present invention provides methods of delivering a nucleic acid to a cell using the AAAV vectors and particles. Methods of isolating the AAAV are provided.
Owner:THE GOVERNMENT OF THE UNITED STATES OF AMERICA AS REPRESENTED BY THE SEC DEPT OF HEALTH & HUMAN SERVICES NAT INST OF HEALTH OFFICE OF TECH TRANSFER +1

Use of the CYP46A1 Gene for the Treatment of Alzheimer's Disease

The invention relates to a viral vector for treating Alzheimers disease, which vector comprises a cholesterol 24-hydroxylase (CYP46A1) encoding nucleic acid. In a preferred embodiment, the viral vector may be an Adeno-Associated-Virus (AAV) vector, preferably an AVV5 vector. The vector may be useful for the manufacture of a pharmaceutical composition for the treatment of Alzheimers disease in a subject, wherein the vector is to be administered directly into the brain of the subject or by intravenous or intrathecal injection.
Owner:INST NAT DE LA SANTE & DE LA RECHERCHE MEDICALE (INSERM)

siRNA for inhibiting expression of human PCSK9 gene and application thereof

The invention discloses an siRNA sequence for specifically inhibiting the expression of a human protein convertase subtilisin 9 (PCSK9) target gene and an application thereof. The siRNA can be chemically synthesized, and can also be constructed into corresponding recombinant lentivirus or adeno-associated virus and the like according to the sequence of the siRNA. After human hepatoma carcinoma cell HepG2 and human normal hepatoma carcinoma cell LO2 are transfected by the siRNA or the virus, the expression of PCSK9 can be effectively inhibited; the protein level of a low-density lipoprotein receptor (LDLR) is increased; and the activity of the hepatoma carcinoma cell for taking in low-density lipoprotein (LDL) is remarkably enhanced. After the recombinant lentivirus is subjected to high-pressure caudal vein injection administration, the levels of total cholesterol (TC) and low-density lipoprotein cholesterol (LDL-C) in plasma of a hypercholesterolemia model mouse can be remarkably reduced. The siRNA or the recombinant virus or a modifier thereof can be used for preventing and / or treating diseases like hyperlipidemia, atherosclerosis, cardiovascular and cerebrovascular diseases, obesity, diabetes and nephropathy.
Owner:CHINA PHARM UNIV

Method for improving expression level of human coagulation factor IX

The invention discloses a method for improving the expression level of human coagulation factor IX. The method comprises the following steps: optimizing human coagulation factor IX gene; replacing Kozak sequence; and constructing AAV (adeno-associated virus) expression plasmid by the optimized human FIX gene and Kozak sequence, and packaging the recombinant AAV. The expression level of the human coagulation factor FIX gene is remarkably improved by the optimized gene coding sequence and Kozak sequence. The optimized human FIX gene and Kozak sequence can be used for efficiently producing FIX protein for treating hemophilia B, and the production cost of the FIX protein is reduced; and the optimized human FIX gene and Kozak sequence can also be used in AAV-mediated gene replacement therapy totreat hemophilia B, the expression level of the FIX gene in human bodies is increased, so that the administration dosage is reduced, and the therapeutic effect is improved.
Owner:SHENZHEN CHANGENE MEDICAL TECH CO LTD

Novel transgenic medaka, gene fragments

The invention also relates to a novel nucleic acid fragment including: (1)medaka beta-actin promotor; (2) a fluoyescent gene; and (3) inverted terminal repeats (ITR) of adeno-associated virus. The invention further relates to a plasmid comprising the nucleic acid fragment of the invention.
Owner:TAIKONG +1

Medical application of encoding gene ESRRA of estrogen-related receptor alpha (ERR alpha)

The invention relates to an application of an encoding gene ESRRA of an estrogen-related receptor alpha (ERR alpha) in preparation of a medicine for preventing or treating liver diseases. The liver diseases include non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), primary biliary cholangitis (PBC), primary sclerosing cholangitis (PSC), alcoholic fatty liver disease (AFLD), hepatic fibrosis, or cirrhosis. The adeno-associated virus is utilized to construct an ERRalpha overexpression vector system, and the fact that the ERR alpha gene and a delivery system of the ERR alpha gene can be used for preventing or treating the liver diseases is proved on various disease models.
Owner:CHINA PHARM UNIV

Adeno-associated virus vector targeting cardiac vascular endothelium and application thereof

The invention belongs to the technical field of biology, and particularly relates to an adeno-associated virus vector for targeting cardiac vascular endothelium and an application of the adeno-associated virus vector. The invention aims to solve the problem that natural AAV has poor cardiovascular endothelial transduction capacity in vivo. The invention provides an adeno-associated virus vector targeting heart vascular endothelium. The library is constructed by inserting an R588 site of an AAV2 capsid protein gene into a coding sequence of random heptapeptide, and inserting the coding sequenceinto a plasmid skeleton containing an AAV2 rep gene and ITR through HindIII / NotI double enzyme digestion. After two rounds of in-vivo directed evolution screening, the two AAV variants EC71 and EC73are obtained, the transduction capacity of AAV to the heart vascular endothelium in vivo is improved, meanwhile, transduction to the liver is greatly reduced, and transgenic expression is maintained for at least four months in the mouse heart vascular endothelium. The EC71 vector is used for conveying the eNOS gene into a myocardial infarction mouse body, so that the activity of the eNOS protein of the heart and the lung is effectively improved, and the EC71 vector has a certain application prospect in gene therapy of cardiovascular endothelial related diseases.
Owner:SICHUAN UNIV

Preparation method of adeno-associated virus with epigenetic modification function and application thereof

The invention relates to an application of a recombinant single-stranded DNA virus vector with a heterologous nucleic acid sequence in preparation of a medicine for treating stress cognitive impairment, which comprises a pharmaceutical composition comprising the virus vector, a method for preparing the virus vector, and an application of the virus vector in research on an epigenetic regulation mechanism and treatment on cognitive disorder caused by stress. Wherein the recombinant single-stranded DNA virus vector is an adeno-associated virus vector, and the heterologous nucleic acid encodes therapeutic protein; wherein the therapeutic protein is TET1 (1418 to 2136aa). The adeno-associated virus with the epigenetic modification function is artificially prepared, expression of neurotrophic factors such as BDNF can be induced through epigenetic modification after the adeno-associated virus infects an organism, neuron survival and neurogenesis are maintained, dendritic spine maturation is promoted, and therefore the effect of resisting and treating stress cognitive injuries is achieved.
Owner:ACADEMY OF MILITARY MEDICAL SCI

Methods for improved therapeutic use of recombinant aav

Provided herein are methods for managing host immune responses to improve therapeutic outcomes in adeno-associated virus (AAV)-mediated gene therapy. Such methods may include administering a recombinant adeno-associated vims (rAAV) to a subject following administration of a CD 19 inhibitor, e.g., an anti-CD 19 antibody. The methods described herein can facilitate improved transgene expression, help overcome pre-existing NAbs, and / or permit redosing with the same or substantially similar rAAV or transgene.
Owner:ULTRAGENYX PHARMA

Optogenetic visual restoration using chrimson

Disclosed are, among other methods, methods for reactivating retinal ganglion cells in mammals by administering an effective amount of channelrhodopsins (such as ChrimsonR), or an effective amount of such channelrhodopsins (such as ChrimsonR) fused to a fluorescent protein, in the form of protein or nucleic acids, and compositions thereof. The methods may include a light stimuli level inducing RGCs response that is below radiation safety limit. The methods may include delivery by an adenoassociated virus vector. The methods may include use of a CAG promoter. The methods may result in a long term expression of an effective amount of the channelrhodopsins (such as ChrimsonR protein).
Owner:GENSIGHT BIOLOGICS SA +2

Adenoassociated virus vectors for the treatment of mucopolysaccharidoses type iv a

The present invention provides new polynucleotide sequences, adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidosis type IVA or Morquio A syndrome.
Owner:ESTEVE PHARMA SA +1

Recombinant gland-related virus AAV-gas6 and application

The invention relates to the technical field of recombinant gland-related viruses and particularly relates to a recombinant gland-related virus AAV-gas6, a preparation method therefor and applicationof the recombinant gland-related virus AAV-gas6. The invention provides the recombinant gland-related virus AAV-gas6 containing a nucleic acid sequence represented by SEQ ID NO: 1. The invention further provides the preparation method for the recombinant gland-related virus AAV-gas6. The recombinant gland-related virus AAV-gas6 provided by the invention is advantageously applied to preparation ofdrugs for treating or relieving JEV-induced viral encephalitis.
Owner:FOURTH MILITARY MEDICAL UNIVERSITY

Method for improving infection efficiency of adeno-associated virus to infect cells

The invention relates to a method for improving infection efficiency of AAV (adeno-associated virus) to infect cells. The method comprises the following steps: making cells to-be-infected contacted with DNA replication inhibitors, wherein the DNA replication inhibitors comprise cell cycle non-specific drugs and / or CDK inhibitors. According to the method disclosed by the invention, the infection efficiency of the AAV to infect cells can be remarkably improved.
Owner:CHIGENOVO CO LTD

Recombinant adeno-associated virus vector, gene composition and application for treating atherosclerosis

The invention provides a recombinant adeno-associated virus vector, gene composition and application of the gene composition for treating atherosclerosis. The recombinant adeno-associated virus vector is obtained by inserting lectin-like oxidized low-density lipoprotein receptor 1 (LOX-1) into the adeno-associated virus vector, and combining it with the TBG promoter to form a gene composition for the preparation of treatment Gene therapy injections for atherosclerosis. The sequence of the LOX-1 gene is shown in SEQ ID NO: 1 in the sequence listing. The present invention utilizes the adeno-associated virus vector to efficiently introduce the drug effect element into the body through intravenous injection, realizes the ectopic high-efficiency expression of the therapeutic effect protein LOX-1 of the drug effect element expression product, and uses the TBG promoter to transfer the drug effect element that is not originally expressed in the liver through the TBG promoter. The LOX-1 receptor is successfully expressed in hepatocytes. With the help of the liver's powerful lipid metabolism and metabolic pathways, it can phagocytose and clear the overloaded OX-LDL in the circulation of patients with atherosclerosis, thereby inhibiting the progression of atherosclerosis.
Owner:XIEHE HOSPITAL ATTACHED TO TONGJI MEDICAL COLLEGE HUAZHONG SCI & TECH UNIV

Adenoassociated virus vectors for the treatment of mucopolysaccharidoses

The present invention provides new adeno-associated virus-derived vectors and pharmaceutical compositions containing the same for the treatment of lysosomal storage disorders and specially, for the treatment of mucopolysaccharidoses Type IIID.
Owner:ESTEVE PHARMA SA +1

Application of poloxamer P338 in improving virus infection efficiency of cells and method thereof

The invention discloses a method for utilizing poloxamer P338 to improve the virus infection efficiency of cells and application thereof. The use of the poloxamer P338 can increase the infection efficiency of lentivirus at low MOI to 293T cells, LLC-MK2 cells and the like. The use of the poloxamer P338 can also promote the infection of adeno-associated virus at the same MOI value to the 293T cellsto enhance the expression of foreign genes carried by viral vectors.
Owner:OBIO TECH SHANGHAI CORP LTD
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