Methods for detecting aav
AssigneeGenzyme Corporation
InventorsXiaoying JIN, Catherine O'Riordan, Lin Liu, Kate Zhang
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Abstract
Provided herein are methods for determining the serotype of a virus particle and/or or determining the heterogeneity of a virus particle (e.g., an AAV particle). In other embodiments, the invention provides methods to determine the heterogeneity of AAV particles. In some aspects, the invention provides viral particles (e.g., rAAV particles) with improved stability and/or improved transduction efficiency by increasing the acetylation and/or deamidation of capsid proteins.
Claims44
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A recombinant AAV (rAAV) particle comprising an amino acid substitution at amino acid residue 2 of VP1 and/or VP3 of a parent polypeptide;
- wherein the amino acid substitution at amino acid residue 2 of VP1 and/or VP3 alters N-terminal acetylation compared to N-terminal acetylation at amino acid residue 2 of VP1 and/or VP3 of the parent AAV particle.
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An AAV capsid protein comprising an amino acid substitution at amino acid residue 2 of a parent AAV capsid protein;
- wherein the amino acid substitution at amino acid residue 2 alters N-terminal acetylation compared to N-terminal acetylation at amino acid residue 2 of the parent AAV capsid protein.
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A method of improving the stability of a rAAV particle, the assembly of rAAV particles in a cell, or the transduction of rAAV particles in a cell, the method comprising substituting amino acid residue 2 of VP1 and/or VP3 of a parent VP1 and/or VP3;
- wherein the substituting the amino acid at position 2 alters N-terminal acetylation of VP1 and/or VP3, as compared to amino acid residue 2 of the parent VP1 and/or VP3.
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Dependencies are inferred from the claim text.