The present invention relates to adeno-associated virus (AAV) vectors modified by the covalent coupling of at least one compound comprising a lactam moiety (e.g., β-lactam) to at least one amino group of an amino acid residue of the capsid of the AAV vectors. The AAV vectors are useful in transducing a cell, especially for gene therapy.

Proposed are a stabilizer for an adeno-associated virus (AAV) and a method for stabilizing an adeno-associated virus using the same. More particularly, the present disclosure relates to a stabilizer for an adeno-associated virus including a surfactant or albumin, an adeno-associated virus liquid formulation including adeno-associated virus the stabilizer, and a production method for an adeno-associated virus with improved stability. The technology, according to the present disclosure, inhibits excessive aggregation between viruses when producing a gene delivery virus and therapeutic nanoparticles using an adeno-associated virus and forming an additional polyphenol binder for this purpose, thereby maintaining the particle size to an effective size for drug delivery and improving the stability in the liquid phase. Therefore, the technology is expected to be very useful in the field of drug delivery technology for gene therapy.

The present disclosure relates generally to methods and adeno-associated virus (AAV) vectors for in vivo transduction. The disclosure is directed to methods for producing modified AAV capsid polypeptides and AAV vectors that are suitable for transduction of cells in vivo, and methods for screening AAV vectors. The disclosure also relates to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules, AAV vectors comprising the capsid polypeptides, and nucleic acid vectors comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.

The objective of the present invention is to provide a method capable of purifying a virus or a virus-like particle easily. The method for purifying a virus or a virus-like particle according to the present invention is characterized in comprising the step of contacting a liquid comprising the virus or the virus-like particle with a water-insoluble inorganic compound, wherein the water-insoluble inorganic compound comprises one or more elements selected from magnesium, calcium and aluminum.

The present invention provides a method of manufacturing vectors containing a heterologous gene-editing protein comprising providing (a) transforming a host system with a nucleic acid cassette containing a promoter operably linked to a gene encoding a gene-editing protein, wherein the host system also contains a heterologous inhibitor for the gene-editing protein, (b) incubating the host system for a time sufficient for vector production and to release the recombinant vector, and (c) recovering the recombinant vector. Also provided herein are cell lines for expressing vectors containing a gene-editing protein with an inhibitor of the gene-editing protein to prevent leaky expression of the gene-editing protein comprising constitutive expression of an inhibitor of a gene-editing protein.

The present disclosure relates generally to modified adeno-associated virus (AAV) from serotypes other than serotype 2, which have a viral capsid protein with a subunit 1 (VP1) sequence which is modified relative to the corresponding wildtype sequence. In particular, the modified AAVs of the disclosure comprise site-specific amino acid substitutions within the phospholipase A2 (PLA2) domain and flanking sequence relative to the corresponding wild-type sequence which improve functionality of the AAV when produced in insect cells. The present disclosure also relates to methods of producing the modified AAVs, reagents therefor, baculovirus expression systems and insect cells for producing said modified AAVs.

The current invention is based, at least in part, on the finding that the transverse nuclear magnetic spin relaxation time T2 and the transverse nuclear magnetic spin relaxation rate R2, respectively, of protons of water molecules in an aqueous solution comprising viral particles depends on the loading status (full vs. empty) of the viral particle. Thus, one aspect of the current invention is a method for determining the ratio of loaded viral particles to empty viral particles in a sample, comprising the steps of determining a nuclear magnetic resonance (NMR) parameter related to the protons of the water molecules present in an aqueous solution comprising a mixture of loaded and empty viral particles by applying an NMR measurement to the solution, and determining the ratio of loaded viral particles to empty viral particles with the NMR parameter determined in the previous step based on a calibration function.

The present disclosure provides a variant AAV capsid protein that confers tropism to lung cells and recombinant adeno-associated viruses comprising the variant AAV and pharmaceutical compositions comprising same and their use in the delivery of heterologous nucleic acids to lung cells for the treatment of pulmonary disorders.

Described herein are compositions and methods for treating Friedreich's Ataxia (FA) using adeno-associated virus (AAV) to deliver therapeutics agents.

The invention relates to chimeric AAV capsids targeted to the central nervous system, virus vectors comprising the same, and methods of using the vectors to target the central nervous system. The invention further relates to chimeric AAV capsids targeted to oligodendrocytes, virus vectors comprising the same, and methods of using the vectors to target oligodendrocytes.