Rational polyploid adeno-associated virus vectors for the treatment of disease
AssigneeThe University of North Carolina at Chapel Hill
InventorsChengwen Li, Richard Jude Samulski
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Abstract
The present invention provides a polyploid adeno-associated virus (AAV) capsid, wherein the capsid comprises capsid protein VP1, wherein said capsid protein VP1 is from one or more than one first AAV serotype, wherein said capsid protein VP2 is from one or more than one first AAV serotype and capsid protein VP3, wherein said capsid protein VP3 is from one or more than one second AAV serotype and wherein at least one of said first AAV serotype is different from at least one of said second AAV serotype and is different from at least one of said third AAV serotype, in any combination.
Claims23
A method of treating a disease or a disorder in a subject, comprising administering to the subject an effective amount of a substantially homogeneous population of AAV virions having at least two viral structural proteins from the group consisting of AAV capsid proteins VP1, VP2, and VP3, wherein the at least two viral structural proteins are sufficient to form an AAV virion that encapsidates an AAV genome, and wherein at least one of the at least two viral structural proteins present is from a single AAV serotype and is from a completely different serotype than the other viral structural protein, and wherein the VP1 is only from one serotype, the VP2 is only from one serotype, and the VP3 is only from one serotype, wherein the AAV virions comprise within their genome a heterologous nucleic acid that encodes a therapeutic protein or a functional RNA for treating the disease or the disorder.
Dependencies are inferred from the claim text.