Abstract: The present disclosure provides compositions and methods for treating neuropathic pain or focal epilepsy. Further provided are AAV capsid polypeptides and/or nucleic acids suitable for transducing neurons related to neuropathic pain or focal epilepsy. The disclosure relates to AAV vectors comprising specific capsid polypeptides that provides desirable transduction efficiency and/or tropism for neurons responsible for neuropathic pain or focal epilepsy management.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells.

Abstract: Provided are variant adeno-associated virus (AAV) capsid proteins and recombinant AAV virions having one or more variant AAV capsid proteins. Also provided are compositions and methods for the use of the recombinant AAV virions, such as for the treatment or prophylaxis of a disease or disorder.

Abstract: Provided is a unit dose of recombinant adeno-associated virus (AAV) particles for expression of matrix metalloproteinase 3 (MMP-3). Further provided is a unit dose of recombinant MMP-3. Also provided are methods of use thereof, e.g., in transducing the corneal endothelium of a subject; reducing intraocular pressure in an eye of a subject; treating and/or preventing elevated intraocular pressure in a subject; and treating and/or preventing glaucoma in a subject. Subjects include primates.

Abstract: Provided are mutant viral capsid cell libraries, individual cells of such libraries, systems, vectors, and methods for generating the cell libraries, and methods of use thereof to screen for mutant viral capsids with desired characteristics.

Abstract: The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein that binds heparan sulfate proteoglycans, where the AAV virions exhibit greater infectivity of retinal cells, altered tropism and/or the ability to bind and cross the inner limiting membrane following intravitreal injection. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors, and methods for the expression of a gene in mammalian cells.

Abstract: The present disclosure provides gene therapy vectors comprising a polynucleotide sequence encoding a LAMP-2 polypeptide, methods of use thereof, pharmaceutical compositions, and more. In particular, the disclosure provides recombinant AAV vectors having AAVrh74 serotype expressing LAMP-2A, LAMP-2B, or LAMP-2C for use as a therapeutic in, for example, Danon Disease.

Abstract: The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g. Danon disease.

Abstract: The present disclosure provides adeno-associated virus (AAV) virions with altered capsid protein that binds heparan sulfate proteoglycans, where the AAV virions exhibit greater infectivity of retinal cells, altered tropism and/or the ability to bind and cross the inner limiting membrane following intravitreal injection. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, and methods of treating ocular disease.

Abstract: The present disclosure relates generally to compositions and methods for treating, preventing, inhibiting, or delaying central nervous system degeneration. The disclosure relates to a recombinant gene therapy vector comprising a PARK2, PINK1, DJ-1, LRRK2, SCNA, c-Rel, ATG7, VMAT2, or GBA gene, or functional fragment or variant thereof. The disclosure also relates to CRISPR/Cas-based gene editing systems for treating, preventing, inhibiting, or delaying central nervous system degeneration.

Abstract: The present disclosure provides modified adeno-associated virus (AAV) virions with altered capsid proteins, where the modified AAV virions exhibit greater infectivity of retinal cells when administered to the eye or greater infectivity of liver cells when administered intravenously. The present disclosure further provides methods of delivering a gene product to a retinal cell in an individual, methods of treating ocular diseases and disorders, methods of delivering a gene product to the liver in an individual, and methods of treating liver diseases and disorders.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors, and methods for the expression of a gene in mammalian cells.

Abstract: Provided are variant adeno-associated virus (AAV) capsid proteins and recombinant AAV virions having one or more variant AAV capsid proteins. Also provided are compositions and methods for the use of the recombinant AAV virions, such as for the treatment or prophylaxis of a disease or disorder.

Abstract: The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g. Danon disease.

Abstract: Provided are mutant viral capsid cell libraries, individual cells of such libraries, systems, vectors, and methods for generating the cell libraries, and methods of use thereof to screen for mutant viral capsids with desired characteristics.

Abstract: The disclosure relates to optimized polynucleotide sequences for LAMP-2B, expression cassettes, vectors, and methods of use thereof in treating disease, e.g. Danon disease.

Abstract: Provided are mutant viral capsid cell libraries, individual cells of such libraries, systems, vectors, and methods for generating the cell libraries, and methods of use thereof to screen for mutant viral capsids with desired characteristics.

Abstract: The present disclosure provides polynucleotide cassettes, expression vectors and methods for the expression of a gene in mammalian cells.