University of California

David Stephen Ojala

David V Schaffer

Philip A Romero

Oscar Westesson

Ian H Holmes

Jorge Santiago-Ortiz

John Weinstein

The present disclosure provides recombinant adeno-associated virus virions with variant capsid protein, where the recombinant AAV (rAAV) virions exhibit one or more of increased ability to cross neuronal cell barriers, increased infectivity of a neural stem cell, increased infectivity of a neuronal cell, and reduced susceptibility to antibody neutralization, compared to a control AAV, and where the rAAV virions comprise a heterologous nucleic acid. The present disclosure provides methods of delivering a gene product to a neural stem cell or a neuronal cell in an individual. The present disclosure also provides methods of modifying a target nucleic acid present in a neural stem cell or neuronal cell.

Adeno-associated virus capsid variants and methods of use thereof

The present disclosure provides recombinant adeno-associated virus (rAAV) virions comprising a variant AAV capsid protein, e.g., an AAV capsid protein derived from an ancestral AAV capsid protein amino acid sequence. An rAAV virion of the present disclosure can exhibit greater infectivity of a target cell. The present disclosure also provides methods of delivering a gene product to a target cell in an individual by administering to the individual an rAAV of the present disclosure. The present disclosure also provides methods of generating rAAV virions that have a variant AAV capsid protein derived from an ancestral AAV capsid protein amino acid sequence.

Adeno-associated virus variants and methods of use thereof

Growing community of inventors

Berkeley, CA, United States of America

David Stephen Ojala