Capsid
There is described an AAV capsid protein having an amino acid sequence which has at least 98% identity to the sequence of SEQ ID NO: 3 or at least 94% identity to the sequence of SEQ ID NO: 4. Also described is a pharmaceutical composition, an AAV capsid and a viral particle comprising the capsid protein, a recombinant AAV vector comprising a nucleotide sequence which encodes for the capsid protein, and a host cell and a transgenic animal comprising the capsid protein or the vector. In addition, there is described a method of transferring a nucleic acid of interest into a mammal comprising introducing a recombinant AAV vector into the mammal, wherein the recombinant AAV vector comprises a gene of interest which is encapsidated into a capsid comprising the capsid protein.
1. A method of delivering a nucleic acid of interest into a liver cell comprising administering intravenously to a human subject a recombinant AAV vector, wherein the recombinant AAV vector comprises an AAV capsid encapsulating a nucleic acid of interest, wherein the AAV capsid comprises AAV capsid proteins, and wherein the AAV capsid proteins have an amino acid sequence which has at least 99% identity to the sequence of SEQ ID NO:3.
2. The method of claim 1 , wherein the AAV capsid proteins have an amino acid sequence which has at least 99.5% identity to the sequence of SEQ ID NO: 3.
3. The method of claim 2 , wherein the AAV capsid proteins have an amino acid sequence that has the sequence of SEQ ID NO:3.
4. The method of claim 1 , wherein the human subject has a disease or disorder.
5. The method of claim 4 , wherein the disease or disorder is congenital FVII deficiency.
6. The method of claim 4 , wherein the disease or disorder is hemophilia.
7. The method of claim 4 , wherein the disease or disorder is Gaucher's disease.
8. The method of claim 4 , wherein the disease or disorder is OTC deficiency.
9. The method of claim 4 , wherein the disease or disorder is Fabry's disease.
10. The method of claim 4 , wherein the disease or disorder is glycogen storage diseases.
11. The method of claim 4 , wherein the disease or disorder is α-1-antitrypsin deficiency.
12. The method of claim 4 , wherein the disease or disorder is progressive familial intrahepatic cholestasis.
13. The method of claim 4 , wherein the disease or disorder is Wilson's disease.
14. The method of claim 4 , wherein the disease or disorder is Crigler Najjar syndrome.
15. The method of claim 4 , wherein the disease or disorder is hepatocellular carcinoma.