12 results about "Adeno associate virus" patented technology

The present disclosure provides modified AAV vectors which express a tripeptidyl peptidase 1 (TPPl) in subject. Aspects of the disclosure provide a modified adeno associated virus 1 (AAV1) vector comprising a capsid protein that comprises a targeting peptide and a nucleic acid molecule comprising a sequence encoding the modified AAV genome comprising a replication (rep) gene, a capsid (cap) gene, and a tripeptidyl peptidase 1 (TPP1) transgene.

Described herein is an immortalized mammalian cell configured for production of a bioproduct of interest, which cell has a safe harbor locus selected from an Adeno Associated Virus 1 (AAVS1) locus, a CCR5 locus, and a Rosa26 locus into which a Human Papillomavirus (HPV) E6 gene sequence and an HPV E7 gene sequence have been inserted. Also disclosed herein are methods for immortalizing a mammalian cell for production of a bioproduct of interest as well as methods of producing a bioproduct of interest.

Disclosed herein are nucleic acid molecules and compositions of preparing recombinant adeno-associated viruses (rAAV) and uses thereof. Specifically, two-plasmid systems are provided for efficient rAAV production.

A HEK293 cell line adapted to serum-free suspension culture and a use thereof. Specifically, the present application relates to a method for preparing a viral vector such as an adeno-associated virus vector using the cell line, and a method for screening the cell line.

Provided are a mutant of an adeno-associated virus (AAV) targeting a resting or activated T cell, a use of the mutant of the AAV, and a heterologous peptide targeting a T cell. An amino acid sequence of the heterologous peptide is set forth in any one of SEQ ID NOS: 1-5. The mutant of the AAV exhibits high efficiency in targeting resting or activated T cells, and has advantages such as low dose, strong infectivity, and high safety. A recombinant adeno-associated virus (rAAV) can be constructed from a mutant of an AAV capsid protein of the present application. Nucleic acid sequences carried by rAAV can not be integrated into the genome of the host cell. T he rAAV can be used for the quick infection and reinfusion of activated T cells, which reduces the unnecessary quality control and in vitro dwell time.

Provided herein are pharmaceutical compositions comprising an adeno-associated virus (AAV). In some aspects, a pharmaceutical composition comprises from about 5 mM to about 20 mM of a Tris buffer, from about 100 mM to about 250 mM of sodium chloride, from about 0.001% (w / v) to about 0.01% (w / v) of Pluronic F68, and has a pH of from 7.0 to 9.0. Also provided are methods of making a pharmaceutical composition comprising the AAV, methods of treating a VEGF-associated condition in a subject, and methods of storing the AAV composition.

The present disclosure provides variant AAV capsid proteins that confer tropism to lung cells, and recombinant adeno-associated viruses comprising the variant AAV capsid proteins, and pharmaceutical compositions comprising the same, and their use in delivering heterologous nucleic acids to lung cells for the treatment of lung conditions.

This disclosure provides methods for identifying a subject suitable for an adeno associated virus (AAV) therapy. In some embodiments, the method comprises measuring a titer of an antibody or antigen-binding portion thereof that specifically binds to an AAV (“anti-AAV antibody”) in a biological sample obtained from the subject using an enzyme-linked immunosorbent assay (ELISA).

Disclosed herein are scientific instrument support systems, as well as related methods, computing devices, and computer-readable media. For example, in some embodiments, a scientific instrument support apparatus may include: first logic to receive, from a cryogenic (Cryo) electron microscopy, cryo-microscopy (Cryo-EM) data regarding a biological specimen having a plurality of capsids; second logic to determine a classification for each of the capsids by processing the Cryo-EM data through an AI model trained under various acquisition conditions; and third logic to display the classifications of the capsids in the biological specimen.

The present disclosure provides an automated method of producing viral vectors, utilizing engineered viral vector-producing cell lines within a fully-enclosed cell engineering system. Exemplary viral vectors that can be produced include lentivirus vectors, adeno-associated virus vectors, baculovirus vectors and retrovirus vectors.

The present disclosure provides modified AAV vectors which express the ε2 allele of apolipoprotein E (ApoE2). in subject. Aspects of the disclosure provide a modified adeno associated virus 1 (AAV1) vector comprising a capsid protein that comprises a targeting peptide and a nucleic acid molecule comprising a sequence encoding the modified AAV genome comprising a replication (rep) gene, a capsid (cap) gene, and an ApoE2 transgene.

The present invention relates to a composition for gene transfer comprising a recombinant virus vector and provides a composition for gene transfer which improves the efficiency of gene transfer using a virus vector. Specifically, the present invention provides a composition for gene transfer, which comprises a recombinant virus vector comprising a gene of interest for expression and a sugar at a concentration of at least 40 mM. The virus vector is preferably an adeno-associated virus vector. By using the composition of the present invention, the efficiency of gene transfer using a virus vector can be improved by about 50 times or more.