48 results about "Adenoassociated virus" patented technology

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.

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.

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.

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.

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.

The present invention relates to promoters that function specifically or preferentially in the liver. These promoters are capable of enhancing liver-specific expression of genes.The invention also relates to expression constructs, vectors and cells comprising such liver- specific promoters, and to methods of their use.The present invention future relates to adeno-associated virus (AAV) gene therapy vectors comprising the liver-specific promoters, therapeutic agents comprising the liver-specific promoters, and methods using the same.

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).

The invention provides a method of treating ovarian cancer in a patient by administering to the patient an adeno-associated virus (AAV) vector encoding an anti-VEGF antibody or antigen binding fragment thereof.

The invention belongs to the field of biomedicine, and relates to a recombinant adeno-associated virus carrying shRNA (shorthairpin RNA, shRNA) with the effect of inhibiting hepatitis B virus, a preparation method thereof and application thereof in preparing medicaments for treating and / or preventing viral diseases. By adopting gene recombination technology, the shRNA with the effect of inhibiting the hepatitis B virus is cloned into skeleton plasmid of an adeno-associated virus vector, and the skeleton plasmid, together with helper plasmid, performs cotransfection on packaging cells to obtain the recombinant adeno-associated virus. The recombinant adeno-associated virus can effectively inhibit the copy and expression of the hepatitis B virus, and has obvious in-vivo inhibition effect, long lasting time and smaller cytotoxicity.

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.

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 effecti ve 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).

The invention provides a method of increasing blood flow or perfusion in an ischemic tissue. The method comprises the steps: inducing angiogenesis, neovascularization or revascularization; increasingskeletal muscle viability; promoting ischemic skin wound healing; treating or preventing gangrene; and / or treating CLI. In various aspects, the method comprises administering to a subject a hybrid adenoassociated virus (AAV) comprising a nucleotide sequence encoding an E-selectin, AAV serotype 2 (AAV2) inverted terminal repeats (ITRs), and a capsid from an AAV other than serotype 2. In various aspects, the method comprises the step of administering to the subject a cell comprising an AAV comprising a nucleotide sequence encoding an E-selectin, AAV2 ITRs, and an AAV2 capsid.

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.

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.