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US 2012/0232133 A1Published application
Delivery of proteins using adeno-associated virus (aav) vectors
AssigneeCalifornia Institute of Technology
InventorsAlejandro Benjamin Balazs, David Baltimore
Life of this patent
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Abstract
Disclosed herein are compositions, systems and methods for delivery of proteins of interest using adeno-associated virus (AAV) vectors.
Claims61
A viral vector, comprising:
- a 5′ inverted terminal repeat (ITR) of adeno-associated virus (AAV) and a 3′ AAV ITR;
- a promoter;
- a restriction site downstream of the promoter to allow insertion of a polynucleotide encoding one or more proteins of interest, and
- a posttranscriptional regulatory element downstream of the restriction site,
- wherein the promoter, the restriction site and the posttranscription regulatory element are located downstream of the 5′ AAV ITR and upstream of the 3′ AAV ITR.
2 more elements
A method for producing a protein of interest in vivo, comprising:
- providing a recombinant adeno-associated virus (AAV) comprising a nucleotide sequence encoding the protein of interest;
- and
- administering the recombinant AAV to the subject, whereby the recombinant AAV expresses the antibody in the subject, wherein the nucleotide is at least about 1.4 kb.
A method for reducing or inhibiting the infection risk of a virus in a subject, comprising:
- providing a recombinant adeno-associated virus (AAV) comprising a nucleotide sequence encoding a neutralizing antibody for the virus;
- and
- administering the recombinant AAV to the subject, whereby the recombinant AAV expresses the antibody in the subject.
A method of producing a recombinant adeno-associated virus (AAV), comprising:
- providing a packaging cell line with a viral construct comprising 5′ AAV inverted terminal repeat (ITR) and 3′ AAV ITR, helper functions for generating a productive AAV infection, and AAV cap genes;
- and
- recovering a recombinant AAV virus from the supernatant of the packaging cell line.
An isolated, synthetic or recombinant polynucleotide, comprising:
- a nucleic acid sequence having at least about 90% or more sequence identity to SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.