# US 8,927,269 B2 · Avian adenoassociated virus and uses thereof

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- Number: US 8,927,269 B2
- Document ID: US8927269B2
- Status: Granted
- Kind: B2
- Application number: 10/557,662
- Filed: 2004-05-18
- Published: 2007-04-26
- Granted: 2015-01-06
- Earliest claimed: 2003-05-19
- Assignees: The United States of America, as represented by the Secretary, Department of Health and Human Services
- Applicants: Ioannis Bossis; John A. Chiorini
- Inventors: Ioannis Bossis; John A. Chiorini
- Web page: https://patentgrep.com/patent/US8927269B2

## CPC

- C12N 7/00: Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof (preparing medicinal viral antigen or antibody compositions, e.g. virus vaccines, A61K39/00)
- A61K 48/0091: {Purification or manufacturing processes for gene therapy compositions}
- C07K 14/005: from viruses
- C12N 15/86: Viral vectors
- C12N 2710/10251: Methods of production or purification of viral material
- C12N 2750/14121: Viruses as such, e.g. new isolates, mutants or their genomic sequences
- C12N 2750/14122: New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
- C12N 2750/14143: viral genome or elements thereof as genetic vector
- C12N: MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA (microbiological testing media C12Q1/00)
- A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D)
- C07K: PEPTIDES (peptides in foodstuffs A23; obtaining protein compositions for foodstuffs, working-up proteins for foodstuffs A23J; preparations for medicinal purposes A61K; peptides containing beta-lactam rings C07D; cyclic dipeptides not having in their molecule any other peptide link than those which form their ring, e.g. piperazine-2,5-diones, C07D; ergot alkaloids of the cyclic peptide type C07D519/02; macromolecular compounds having statistically distributed amino acid units in their molecules, i.e. when the preparation does not provide for a specific; but for a random sequence of the amino acid units, homopolyamides and block copolyamides derived from amino acids C08G69/00; macromolecular products derived from proteins C08H1/00; preparation of glue or gelatine C09H; single cell proteins, enzymes C12N; genetic engineering processes for obtaining peptides C12N15/00; compositions for measuring or testing processes involving enzymes C12Q; investigation or analysis of biological material G01N33/00)

## Abstract

The present invention provides an Avian adeno-associated virus (AAAV) virus and vectors and particles derived therefrom. In addition, the present invention provides methods of delivering a nucleic acid to a cell using the AAAV vectors and particles. Methods of isolating the AAAV are provided.

## Claims

### Claim 1

A nucleic acid vector comprising:
a) a pair of inverted terminal repeats (ITRs), each of which is capable of forming a T-shaped hairpin structure, and wherein at least one inverted terminal repeat (ITR) comprises SEQ ID NO:20 and SEQ ID NO:21; and,
b) a heterologous nucleic acid sequence between the inverted terminal repeats,
wherein at least one ITR can be used as an origin of replication.

### Claim 2 (depends on 1)

The nucleic acid vector of claim 1, wherein said inverted terminal repeats independently comprise a nucleic acid sequence at least 70% identical to SEQ ID NO:16 or SEQ ID NO:17.

### Claim 3 (depends on 1)

The nucleic acid vector of claim 1, wherein said inverted terminal repeats independently comprise a nucleic acid sequence at least 90% identical to SEQ ID NO:16 or SEQ ID NO:17.

### Claim 4 (depends on 1)

The nucleic acid vector of claim 1, wherein each of said inverted terminal repeats independently comprises SEQ ID NO: 16 or SEQ ID NO: 17.

### Claim 5 (depends on 1)

The nucleic acid vector of claim 1, wherein said heterologous nucleic acid sequence is functionally linked to a promoter.

### Claim 6 (depends on 1)

The nucleic acid vector of claim 1, wherein said heterologous nucleic acid sequence is functionally linked to a promoter comprising SEQ ID NO:24.

### Claim 7

A recombinant AAAV virion comprising
a) a pair of inverted terminal repeats (ITRs), each of which is capable of forming a T-shaped hairpin structure, and wherein at least one inverted terminal repeat comprises SEQ ID NO:20 and SEQ ID NO:21; and,
b) a heterologous nucleic acid sequence between the inverted terminal repeats,
wherein at least one ITR can be used as an origin of replication.

### Claim 8 (depends on 7)

The recombinant AAAV virion of claim 7, wherein said inverted terminal repeats independently comprise a nucleic acid sequence at least 70% identical to SEQ ID NO:16 or SEQ ID NO:17.

### Claim 9 (depends on 7)

The recombinant AAAV virion of claim 7, wherein said inverted terminal repeats independently comprise a nucleic acid sequence at least 90% identical to SEQ ID NO:16 or SEQ ID NO:17.

### Claim 10 (depends on 7)

The recombinant AAAV virion of claim 7, wherein each of said inverted terminal repeats independently comprises SEQ ID NO: 16 or SEQ ID NO: 17.

### Claim 11 (depends on 7)

The recombinant AAAV virion of claim 7, wherein said heterologous nucleic acid sequence is functionally linked to a promoter.

### Claim 12 (depends on 7)

The recombinant AAAV virion of claim 7, wherein said heterologous nucleic acid sequence is functionally linked to a promoter comprising SEQ ID NO:24.

### Claim 13

A vector comprising a nucleic acid sequence selected from the group consisting of SEQ ID NO:10, SEQ ID NO:12 and SEQ ID NO:14, wherein the vector is a plasmid, a yeast artificial chromosome or a non-AAV viral vector.

### Claim 14

A vector that encodes a protein comprising SEQ ID NO:11, SEQ ID NO: 13 or SEQ ID NO: 15, wherein the vector is a plasmid, a yeast artificial chromosome or a non-AAV viral vector.

### Claim 15

A vector system for producing infectious AAAV particles, said vector system comprising at least one nucleic acid molecule selected from the group consisting of:
(a) a nucleic acid molecule comprising a pair of inverted terminal repeats (ITRs), each of which is capable of forming a T-shaped hairpin structure, and a heterologous nucleic acid sequence between the inverted terminal repeats, wherein at least one inverted terminal repeat comprises SEQ ID NO:20 and SEQ ID NO:21, and wherein at least one ITR can be used as an origin of replication; and,
(b) a recombinant nucleic acid molecule that encodes a protein comprising SEQ ID NO:11, SEQ ID NO:13 or SEQ ID NO:15, wherein the recombinant nucleic acid molecule is a plasmid, a yeast artificial chromosome or a non-AAV vector.

### Claim 16 (depends on 15)

The vector system of claim 15, wherein said inverted terminal repeats independently comprise a nucleic acid sequence at least 70% identical in sequence to SEQ ID NO: 16 or SEQ ID NO: 17.

### Claim 17 (depends on 15)

The vector system of claim 15, wherein said inverted terminal repeats independently comprise a nucleic acid sequence at least 90% identical in sequence to at least one sequence selected from SEQ ID NO:16 and SEQ ID NO:17.

### Claim 18 (depends on 15)

The vector system of claim 15, wherein each of said inverted terminal repeats independently comprises SEQ ID NO: 16 or SEQ ID NO: 17.

### Claim 19 (depends on 15)

The vector system of claim 15, wherein said heterologous nucleic acid sequence is functionally linked to a promoter.

### Claim 20 (depends on 15)

The vector system of claim 15, wherein said encoded protein capable of forming infectious AAAV particles comprises an amino acid sequence selected from the group consisting of SEQ ID NO:11, SEQ ID NO:13 and SEQ ID NO:15.

### Claim 21

A nucleic acid vector comprising:
(a) a nucleic acid sequence comprising a pair of inverted terminal repeats (ITRs) flanking a heterologous nucleic acid sequence, each inverted terminal repeat being capable of forming a T-shaped hairpin structure, wherein at least one inverted terminal repeat comprises SEQ ID NO:20 and SEQ ID NO:21, and wherein at least one ITR can be used as an origin of replication;
(b) a nucleic acid sequence encoding a protein comprising SEQ ID NO:11, SEQ ID NO:13 or SEQ ID NO:15; and
(c) a nucleic acid sequence encoding a protein comprising an amino acid sequence selected from the group consisting of SEQ ID NO:3, SEQ ID NO:5, SEQ ID NO:7 and SEQ ID NO:9.