Abstract: Provided for herein are polynucleotides, including codon-optimized polynucleotides, encoding genes for use in, for example, virally-mediated gene therapy for Friedreich's ataxia. Some embodiments related to viral vector constructs for use in such gene therapy. Also provided are dosing regimens and therapeutic combinations or systems for use in modulating immune responses to such viral vectors.

Abstract: The present disclosure relates to compositions and methods for the treatment of TECPR2-associated diseases or disorders. Several embodiments provided for herein relate to virally-mediated transfer of a gene to target cells to induce expression of an encoded polypeptide, protein or other product in order to ameliorate one or more symptoms of a TECPR2-associated disease or disorder in a subject. In several embodiments, the disclosed methods and compositions relate to recombinant adeno-associated virus particles encoding human TECPR2 in order to treat TECPR2-associated diseases or disorders, including spastic paraplegic type 49 (SPG49) and/or hereditary sensory and autonomic neuropathy 9 (HSAN9).

Abstract: Provided herein are methods related to co-packaging of multiple rAAV particles, e.g., by introducing multiple nucleic acid vectors encoding proteins or polypeptides or RNAs of interest into a single cell preparation.

Abstract: Provided herein are methods related to co-packaging of multiple rAAV particles, e.g., by introducing multiple nucleic acid vectors encoding proteins or polypeptides or RNAs of interest into a single cell preparation.

Abstract: Provided herein are modified AAV capsid proteins, particles, nucleic acid vectors, and compositions thereof, as well as methods of their use.

Abstract: Provided herein are compositions, kits and methods related to permitting a human or non-human primate subject to receive multiple doses of recombinant adeno-associate virus (rAAV) vectors. In some aspects, provided herein are methods comprising administering an anti-CD20 antibody, optionally in combination with an mTOR inhibitor, to the subject to permit multiple doses of an rAAV vector to be administered.

Abstract: Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve us of a FXN coding sequence, a truncated FXN 3? UTR, and a prompter.

Abstract: Provided herein are methods of improving rAAV production in cells, the method comprising increasing the salt concentration in the media in which the cells are infected or transfected, cultured, or in which they produce AAV. Aspects of the disclosure relate to improved methods of rAAV production by co-infecting suspension adapted cells (e.g., suspension adapted HEK293 cells) with viruses that encode one or more AAV components for producing rAAV particles within the suspension adapted cells.

Abstract: The present invention provides compositions and methods of use pertaining to rAAV-mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.

Abstract: Provided herein are methods of improving rAAV production in cells, the method comprising increasing the salt concentration in the media in which the cells are infected or transfected, cultured, or in which they produce AAV. Aspects of the disclosure relate to improved methods of rAAV production by co-infecting suspension adapted cells (e.g., suspension adapted HEK293 cells) with viruses that encode one or more AAV components for producing rAAV particles within the suspension adapted cells.

Abstract: Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve us of a FXN coding sequence, a truncated FXN 3? UTR, and a prompter.

Abstract: Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve use of a FXN coding sequence, a truncated FXN 3? UTR, and a promoter.

Abstract: The present invention provides compositions and methods of use pertaining to rAAV-mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.

Abstract: Provided herein are methods related to co-packaging of multiple rAAV particles, e.g., by introducing multiple nucleic acid vectors encoding proteins or polypeptides or RNAs of interest into a single cell preparation.

Abstract: The present invention provides compositions and methods of use pertaining to rAAV-mediated delivery of therapeutically effective molecules for treatment of diseases such as Pompe disease. These compositions in combination with various routes and methods of administration result in targeted expression of therapeutic molecules in specific organs, tissues and cells.

Abstract: Provided herein are methods related to co-packaging of multiple rAAV particles, e.g., by introducing multiple nucleic acid vectors encoding proteins or polypeptides or RNAs of interest into a single cell preparation.

Abstract: Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve us of a FXN coding sequence, a truncated FXN 3? UTR, and a prompter.

Abstract: Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve use of a FXN coding sequence, a truncated FXN 3? UTR, and a promoter.

Abstract: Provided herein are compositions and methods related to purification of recombinant adeno-associated virus (rAAV) particles.

Abstract: Provided herein are nucleic acids, recombinant adeno-associated viral particles, compositions and methods related to treating Friedreich's ataxia. In some examples, the nucleic acids, recombinant adeno-associated viral particles, compositions and methods involve use of a FXN coding sequence, a truncated FXN 3? UTR, and a promoter.