Modified adeno-associated viral vectors for use in genetic engineering
AssigneeIntima Bioscience, Inc.
InventorsModassir CHOUDHRY, Mavis AGBANDJE-MCKENNA, Tilmann BUERCKSTUEMMER, Thomas HENLEY, Lydia VINEY
Life of this patent
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Abstract
Adeno-associated virus has numerous advantages for its use in gene therapy. The present disclosures provide genetically modified adeno-associated viral vectors, and the methods of making the genetically modified adeno-associated viral vectors and compositions in treating cancer, other conditions, diseases, and disorders.
Claims27
A polynucleic acid sequence that encodes:
- a. in a first reading frame, an adeno-associated virus (AAV) VP1 polypeptide, an AAV VP2 polypeptide, and an AAV VP3 polypeptide, and
- b. in a second reading frame, a modified AAV assembly-activating protein (AAP) polypeptide that is at least partially in a region of said first reading frame that encodes at least a portion of said VP2 polypeptide and at least a portion of said VP3 polypeptide, and
- wherein said AAP polypeptide comprises i) at least one amino acid substitution in said region of said first reading frame that encodes at least a portion of said VP2 polypeptide as compared to a wild-type AAV AAP polypeptide of the same AAV serotype of said VP2 polypeptide;
- or ii) at least one amino acid substitution in said region of said first reading frame that encodes at least a portion of said VP3 polypeptide as compared to a wild-type AAV AAP polypeptide of the same AAV serotype of said VP3 polypeptide,
- and wherein one of said VP1, VP2, and VP3 polypeptides is a first AAV serotype, and one of said VP1, VP2, and VP3 polypeptides is a second AAV serotype, wherein said first and second AAV serotypes are different.
(canceled)
-6. (canceled)
-16. (canceled)
A polynucleic acid sequence that encodes i) in a first reading frame, a VP2 polypeptide of an AAV serotype, and ii) in a second reading frame, a modified assembly-activating protein (AAP) polypeptide comprising at least one amino acid substitution within amino acids 5-40 in said modified AAP polypeptide with respect to a wild type AAP polypeptide of the AAV serotype.
-29. (canceled)
-67. (canceled)
A system comprising a first polynucleic acid sequence that encodes at least three adeno-associated virus (AAV) polypeptides, wherein said first polynucleic acid sequence encodes a VP1 polypeptide, a VP2 polypeptide, and a VP3 polypeptide, wherein two of said VP1, VP2, and VP3 polypeptides are from a first AAV serotype, and one of said VP1, VP2, and VP3 polypeptides is from a second AAV serotype, wherein said first AAV serotype and said second AAV serotype are not the same;
- and a second polynucleic acid sequence heterologous to said first polynucleic acid sequence that encodes an AAV assembly-activating protein (AAP) polypeptide, wherein said first polynucleic acid sequence and second polynucleic acid sequence are not covalently linked.
-70. (canceled)
-111. (canceled)
Dependencies are inferred from the claim text.