Methods of purifying AAV particles using a chromatin-DNA nuclease (e.g., MNase), and compositions that include AAV particles and a chromatin-DNA nuclease (e.g., MNase) are described. Compositions and kits that include a chromatin-DNA nuclease (e.g., MNase) for the purification of AAV particles are also provided.

A variant AAV capsid protein, comprising an amino acid sequence corresponding to the position amino acids 585 to 597 or 598 of AAV8 or the position amino acids 583 to 595 or 596 of AAV9, and the polynucleotide, host cells, vectors, AAV virion or the pharmaceutical composition thereof. A method of treating disease, the method comprising administering to a subject in need thereof an effective amount of the recombinant AAV virion.

Provided herein are compositions that include one or more adeno-associated virus (AAVs) vectors and methods of inducing differentiation of a hair cell using these vector(s).

The technology described herein provides variant adeno-associated viral capsid polypeptides and viruses comprising the same. Further provided herein are methods for delivering a viral payload using viruses comprising variant capsid polypeptides described herein. Described herein are viral vectors comprising a variant sequence of the capsid gene, VP1. In particular, viral vectors with capsid polypeptide mutations that modify tropism of the viral particles relative to particles with wild-type capsid polypeptide are described.

The present invention relates to synthetic retinal ON-bipolar cell-specific promoter sequences and their use in therapeutic transgene delivery to the eye for the improvement and/or restoration of vision. The invention features metabotropic glutamate receptor 6 (mGluR6) promoters for an increased and more specific expression in ON-bipolar cells, in particular in cone ON-bipolar cells of the human macula.

The present invention relates to transcription regulatory elements (TREs) such as promoters, which may be used to express a transgene within a cell such as a mammalian cell. The invention further relates to polynucleotides and vectors comprising such transcription regulatory elements, which may be operably linked to a transgene, as well as methods of gene therapy based on using such vectors.

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.

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

The present invention relates to a mutated parvovirus structural protein, comprising at least one insertion comprising a sequence of at least six consecutive amino acids comprised within amino acids 320 to 641 of human HSP70i. Furthermore, the invention relates to multimeric structures comprising the protein, VLPs, a method of producing the mutated parvovirus structural protein and to medicaments or vaccines comprising the mutated parvovirus structural protein that may be used for treating vitiligo or other autoimmune diseases.

The disclosure provides compositions and methods for the preparation, manufacture and therapeutic use of viral vectors, such as adeno-associated virus (AAV) particles having viral genomes encoding one or more antibodies or antibody fragments or antibody-like polypeptides, for the prevention and/or treatment of diseases and/or disorders.