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US 12,509,705 B2Granted
AAV vector variants for ocular gene delivery
AssigneesUniversitäT Bern, Medizinische Hochschule Hannover
InventorsSonja Kleinlogel, Anand David, Hildegard Büning
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Abstract
The present invention relates to adeno-associated virus capsid polypeptide sequences and their use in therapeutic transgene delivery to the eye and potentially other tissues.
Claims21
An adeno-associated virus (AAV) capsid polypeptide comprising a peptide insert in the C-terminal direction at position 453 or in the C-terminal direction at position 587, 588, 589, 590, 591 or 592 of the AAV serotype 2 capsid having amino acid sequence of SEQ ID NO:
- 1 or a position homologous thereto in an AAV of another serotype, wherein the peptide insert is selected from:
- (Cap3;
- SEQ ID NO:
- 10)
- SASEAST,
- (Cap5;
- SEQ ID NO:
- 11)
- DTRPHDQ,
- (Cap7;
- SEQ ID NO:
- 12)
- EHYNSTC,
- (Cap9;
- SEQ ID NO:
- 13)
- PNPNCTL,
- (Cap11;
- SEQ ID NO:
- 14)
- TPPSITA,
- (Cap12;
- SEQ ID NO:
- 15)
- CGESSYL,
- (Cap13;
- SEQ ID NO:
- 16)
- PRTPHTA
- and
- (Cap14;
- SEQ ID NO:
- 17)
- ELCDGFA.
31 more elements
Dependencies are inferred from the claim text.