Adeno-associated virus vectors and methods of use thereof
AssigneeThe Children's Hospital of Philadelphia
InventorJohn H. Wolfe
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Abstract
The present invention provides AAV vectors and methods of use thereof for delivery of transgenes or therapeutic nucleic acids to subjects.
Claims18
A method for delivering a nucleic acid molecule to the brain of a subject, said method comprising administering an adeno-associated virus (AAV) vector to said subject, wherein said AAV vector comprises said nucleic acid molecule and comprises hu.32 capsid protein or rh.8 capsid protein.
A method for treating a disease or disorder affecting the brain of a subject, said method comprising administering an adeno-associated virus (AAV) vector to said subject, wherein said AAV vector comprises a nucleic acid molecule encoding a therapeutic protein or inhibitory nucleic acid molecule and comprises hu.32 capsid protein or rh.8 capsid protein.
Dependencies are inferred from the claim text.