48 results about "Adenoassociated virus" patented technology

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.

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.

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.