Abstract: This invention is directed to AAV compositions for circular RNA expression and methods of expressing covalently closed, circular RNA.

Abstract: Disclosed herein are compositions comprising one or more subgenomic Flavivirus RNA (sfRNA) elements and using those compositions in methods of improving mRNA transcript stability, improving mRNA transcript translation efficiency, enhancing gene therapy, and treating and/or preventing a disease or disorder.

Abstract: Disclosed herein are compositions for and methods of generating chimeric RNA molecules and methods of treating and/or preventing a genetic disease or disorder using chimeric RNA molecules.

Abstract: The present disclosure provides compositions for and methods of preventing protein aggregation, removing protein aggregates, and improving fluid flux in the brain.

Abstract: The present disclosure provides adeno-associated virus (AAV) vectors, comprising coevolved capsid variant proteins, pharmaceutical compositions, methods of making, and methods for delivering such to a subject.

Abstract: The present disclosure provides nucleic acid expression cassettes, vectors, compositions and methods for the treatment of ATPase-mediated diseases in a subject.

Abstract: The present disclosure provides adeno-associated virus (AAV) vectors, comprising coevolved capsid variant proteins, pharmaceutical compositions, methods of making, and methods for delivering such to a subject.

Abstract: The present disclosure provides AAV capsid proteins comprising a modification in the amino acid sequence and virus capsids and virus vectors comprising the modified AAV capsid protein. The disclosure also provides methods of administering the virus vectors and virus capsids of the disclosure to a cell or to a subject in vivo.

Abstract: The present invention provides AAV capsid proteins comprising a modification in the amino acid sequence and virus vectors comprising the modified AAV capsid protein. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject in vivo.

Abstract: Disclosed herein are purification matrices comprising adeno-associated virus (AAV)-binding polypeptides and methods of using the same.

Abstract: The present disclosure provides nucleic acid expression cassettes, vectors, compositions and methods for the treatment of ATPase-mediated diseases in a subject.

Abstract: This invention is directed to AAV compositions for circular RNA expression and methods of expressing covalently closed, circular RNA.

Abstract: The present disclosure provides AAV capsid proteins comprising a modification in the amino acid sequence and virus vectors comprising the modified AAV capsid protein. The disclosure also provides methods of administering the virus vectors and virus capsids of the disclosure to a cell or to a subject in vivo. This disclosure also relates to recombinant adeno associated virus (rAAV) capsid proteins comprising one or more amino acid substitutions in the 5-fold region of the rAAV capsid that result in decreased reactivity to neutralizing antibodies, and their use as gene delivery vehicles.

Abstract: This invention is directed to AAV compositions for circular RNA expression and methods of expressing covalently closed, circular RNA.

Abstract: The present disclosure provides adeno-associated virus (AAV) vectors, comprising coevolved capsid variant proteins, pharmaceutical compositions, methods of making, and methods for delivering such to a subject.

Abstract: The present disclosure provides nucleic acid molecules encoding for at least two circular RNA (circRNAs), adeno-associated virus (AAV) particles including nucleic acid molecules encoding for at least two circRNAs, pharmaceutical compositions, and methods for delivering such to a subject.

Abstract: Adult form glycogen storage disease type III (GSD III) is an orphan neuromuscular disorder caused by a deficiency of glycogen debranching enzyme. Long-term complications include progressive liver fibrosis, hepatic failure, and end-stage liver cirrhosis, and progressive muscle myopathy. Presently, there are no clinically approved therapies or cures for GSD III. Disclosed herein are compositions for and methods of treating and/or preventing GSD III disease progression.

Abstract: The present disclosure provides, in part, compositions and methods for transient removal of neutralizing antibodies directed to AAV vectors. Such compositions and methods expand the patient cohort eligible for gene therapy and also for redosing/re-administration of AAV in patients previously treated with AAV vectors.

Abstract: This invention is directed to AAV compositions for circular RNA expression and methods of expressing covalently closed, circular RNA.

Abstract: The present invention provides AAV capsid proteins comprising a modification in the amino acid sequence and virus vectors comprising the modified AAV capsid protein. The invention also provides methods of administering the virus vectors and virus capsids of the invention to a cell or to a subject in vivo.