Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids
AssigneeThe Trustees of the University of Pennsylvania
InventorsMarkus Hildinger, James M. Wilson, Alberto Auricchio
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Abstract
A pseudotyped rAAV is described, which contains sequences derived from AAV5. A method for producing rAAV pseudotyped with AAV5 capsid is described in which the rep proteins of the serotype or any cross-reactive serotype of the AAV ITRs are utilized. A similar method may be provided to generate a pseudotyped rAAV in which minigenes carrying AAV5 ITRs are packaged in capsids of a heterologous AAV serotype. Also provided are pharmaceutical compositions containing the pseudotyped rAAV and methods of using them for gene delivery.
Claims6
A method of delivering a heterologous molecule to a selected mammalian or veterinary patient, comprising:
- contacting the cells of the patient with a recombinant type 5 adeno-associated virus (rAAV5), said rAAV5 comprising:
- a minigene comprising AAV 5′ ITRs, a heterologous nucleic acid molecule, wherein said nucleic acid molecule is a transgene comprising a nucleic acid sequence encoding a product which is under the control of regulatory sequences which direct expression of the product in a host cell, and AAV 3′ ITRs;
- an AAV5 capsid in which said transgene is packaged;
- wherein said minigene comprises AAV ITR sequences from a serotype heterologous to AAV5, and
- contacting the cells of the patient with a second rAAV, wherein the second rAAV comprising AAV 5′ ITRs, a transgene comprising a nucleic acid sequence encoding a product which is under the control of regulatory sequences which direct expression of the product in a host cell, and AAV 3′ ITRs, packaged in an AAV capsid which is from a serotype different from that of the rAAV5 capsid.
Dependencies are inferred from the claim text.