Adeno-associated virus capsid polypeptides and vectors
AssigneesChildren's Medical Research Institute, The Sydney Children's Hospitals Network (Randwick and Westmead) (Incorporating the Royal Alexandra Hospital for Children), LogicBio Therapeutics, Inc.
InventorsLeszek Lisowski, Marti Cabanes Creus, Ian Alexander, Matthias Charles Jerome Hebben
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Abstract
The present disclosure relates generally to adeno-associated virus (AAV) capsid polypeptides and encoding nucleic acid molecules. The disclosure also relates to AAV vectors comprising the capsid polypeptides, and nucleic acid vectors (e.g. plasmids) comprising the encoding nucleic acids molecules, as well as to host cells comprising the vectors. The disclosure also relates to methods and uses of the polypeptides, encoding nucleic acids molecules, vectors and host cells.
Claims11
A capsid polypeptide, comprising:
- (i) the sequence of amino acids set forth in SEQ ID NO:
- 12, or a sequence having at least 99% sequence identity thereto;
- (ii) the sequence of amino acids at positions 138-735 of SEQ ID NO:
- 12;
- or a sequence having at least 99% sequence identity thereto;
- and/or
- (iii) the sequence of amino acids at positions 204-735 of SEQ ID NO:
- 12;
- or a sequence having at least 99% sequence identity thereto.
A capsid polypeptide, comprising:
- (i) the sequence of amino acids set forth in SEQ ID NO:
- 12 or a sequence having at least 95% sequence identity thereto;
- (ii) the sequence of amino acids at positions 138-735 of SEQ ID NO:
- 12 or a sequence having at least 95% sequence identity thereto;
- and/or
- (iii) the sequence of amino acids at positions 204-735 of SEQ ID NO:
- 12 or a sequence having at least 95% sequence identity thereto;
- wherein the capsid polypeptide comprises:
- a) the sequence of amino acids ISSQSGASNDNH (SEQ ID NO:80) at positions 261-272, with numbering relative to SEQ ID NO:
- 12;
- and
- b) the sequence of amino acids KTGATNKTTLENVLMTNEEEIRP (SEQ ID NO:81) at positions 545-567, with numbering relative to SEQ ID NO:
- 12 and the sequence of amino acids SSNLQAANTAAQTQVVNN (SEQ ID NO:60) at positions 580-597, with numbering relative to SEQ ID NO:
- 12.
Dependencies are inferred from the claim text.