Metabolically activated recombinant viral vectors and methods for their preparation and use
DCFirst Claim
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1. A composition comprising a purified recombinant adeno-associated virus (rAAV) particle comprising an AAV capsid and a single-stranded rAAV vector genome, wherein the rAAV vector genome comprises in the 5′
- to 3′
direction;
a 5′
AAV inverted terminal repeat (ITR) sequence, a first heterologous nucleotide sequence, an internal AAV ITR sequence, a second heterologous nucleotide sequence, and a 3′
AAV ITR sequence, wherein the first heterologous nucleotide sequence can form intrastrand base pairs with the second nucleotide sequence along most or all of its length.
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Abstract
Recombinant viral vectors, especially parvovirus vectors such as adeno-associated virus (AAV) vectors; capable of enhanced expression of heterologous sequences, and methods for their construction and use, are provided. The vectors have a structure, or are capable of rapidly adopting a structure, which involves intrastrand base pairing of at least one region in a heterologous sequence.
41 Citations
36 Claims
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1. A composition comprising a purified recombinant adeno-associated virus (rAAV) particle comprising an AAV capsid and a single-stranded rAAV vector genome, wherein the rAAV vector genome comprises in the 5′
- to 3′
direction;
a 5′
AAV inverted terminal repeat (ITR) sequence, a first heterologous nucleotide sequence, an internal AAV ITR sequence, a second heterologous nucleotide sequence, and a 3′
AAV ITR sequence, wherein the first heterologous nucleotide sequence can form intrastrand base pairs with the second nucleotide sequence along most or all of its length. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18)
- to 3′
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19. A method of expressing a polynucleotide coding sequence in a cell, comprising subjecting the cell to conditions which allow expression of the coding sequence, wherein the coding sequence is introduced into the cell by contacting the cell essentially in the absence of an AAV helper virus with a composition comprising a purified recombinant adeno-associated virus (rAAV) particle, wherein the rAAV particle comprises an AAV capsid and a single-stranded rAAV vector genome, wherein the rAAV vector genome comprises in the 5′
- to 3′
direction;
a 5′
AAV inverted terminal repeat (ITR) sequence, a first heterologous nucleotide sequence, an internal AAV ITR sequence, a second heterologous nucleotide sequence, and a 3′
AAV ITR sequence, wherein the first heterologous nucleotide sequence can form intrastrand base pairs with the second nucleotide sequence along most or all of its length, and wherein the first or the second heterologous nucleotide comprises the coding sequence. - View Dependent Claims (20, 21, 22, 23, 24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35, 36)
- to 3′
Specification