44 results about "Adenoassociated virus" patented technology

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.

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).

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.

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 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.

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.

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.

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.

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.

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.

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 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.

The invention provides a method of increasing blood flow or perfusion in an ischemic tissue; inducing angiogenesis, neovascularization or revascularization; increasing skeletal 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 administering to the subject a cell comprising an AAV comprising a nucleotide sequence encoding an E-selectin, AAV2 ITRs, and a AAV2 capsid.