Adeno-associated virus capsid proteins
AssigneeBiomarin Pharmaceutical Inc.
InventorsPeter Cameron COLOSI, Michael LOCHRIE, Robert NG
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Abstract
The invention relates to novel adeno-associated virus (AAV) capsid proteins, AAV particles comprising a novel capsid protein, polynucleotides encoding these capsid proteins and AAV vectors expressing these capsid proteins. The invention also relates to methods of making the herein described AAV vectors expressing the novel capsid proteins of the invention and associated therapeutic uses of thereof.
Claims52
An adeno-associated virus (AAV) comprising a capsid protein, wherein the capsid protein comprises an amino acid sequence that is at least 95% identical to (i) any one of SEQ ID NOS:15-89, (ii) the VP2 region of any one of SEQ ID NOS:
- 15-89, or (iii) the VP3 region of any one of SEQ ID NOS:
- 15-89, and further comprising a transgene comprising a heterologous gene operably linked to regulatory sequences that control expression of the heterologous gene in a host cell.
A method of treating a subject suffering from a disorder or disease associated with abnormal activity of an endogenous protein comprising administering to the subject an effective amount of an AAV comprising a capsid protein, wherein the capsid protein comprises an amino acid sequence that is at least 95% identical to (i) any one of SEQ ID NOS:15-89, (ii) the VP2 region of any one of SEQ ID NOS:
- 15-89, or (iii) the VP3 region of any one of SEQ ID NOS:15-89, and wherein the AAV comprises a transgene encoding a biologically active copy of the protein.
A vector comprising a nucleic acid sequence encoding an adeno-associated virus (AAV) capsid protein having an amino acid sequence that is at least 95% identical to (i) any one of SEQ ID NOS:15-89, (ii) the VP2 region of any one of SEQ ID NOS:15-89, or (iii) the VP3 region of any one of SEQ ID NOS:
- 15-89.
An adeno-associated virus (AAV) comprising a chimeric capsid protein having a VP1 amino acid sequence of a recipient backbone AAV capsid comprising variable regions I, II, III, IV, V, VI, VII, VIII and IX, except wherein one or more of variable regions I, II, III, IV, V, VI, VII, VIII, or IX is replaced by the corresponding variable region from one or more donor AAV capsids.
A vector comprising a nucleic acid sequence encoding a chimeric adeno-associated virus (AAV) capsid protein, wherein the chimeric AAV capsid protein comprises a VP1 amino acid sequence of a recipient AAV capsid sequence having variable regions I, II, III, IV, V, VI, VII, VIII and IX, and wherein one or more of variable regions I, II, III, IV, V, VI, VII, VIII, or IX is replaced by the corresponding variable region from a donor AAV capsid sequence, wherein the amino acid sequences of the chimeric AAV capsid protein and the recipient capsid sequence are different.
A cDNA comprising a nucleic acid sequence encoding a a chimeric capsid protein having a VP1 amino acid sequence of a recipient backbone AAV capsid comprising variable regions I, II, III, IV, V, VI, VII, VIII and IX, and wherein one or more of variable regions I, II, III, IV, V, VI, VII, VIII, or IX is replaced by the corresponding variable region from one or more donor AAV capsids.
Dependencies are inferred from the claim text.