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US 12,735,725 B2Granted
Altering tissue tropism of adeno-associated viruses
AssigneesMassachusetts Eye and Ear Infirmary, The Schepens Eye Research Institute, Inc.
InventorsLuk H. Vandenberghe, Pauline Schmit, Christopher Tipper, Carmen Unzu, Eric Zinn
Life of this patent
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Abstract
This disclosure provides compositions and methods for altering or changing the tissue tropism, e.g., liver tropism, of adeno-associated viruses (AAV).
Claims6
An adeno-associated virus (AAV) comprising a capsid protein, which comprises the amino acid sequence as set forth in:
- a) SEQ ID NO:87, which corresponds to an AAV9 capsid protein comprising a modification in its liver toggle site to comprise AAV3B-VR1 (SQSGASNDN, SEQ ID NO:
- 58);
- b) SEQ ID NO:88, which corresponds to an AAV80L65 capsid protein comprising a modification in its liver toggle site to comprise AAV3B-VR1 (SQSGASNDN, SEQ ID NO:
- 58);
- c) SEQ ID NO:89, which corresponds to an AAV80L65 capsid protein comprising a modification in its liver toggle site to comprise AAV9-VR1 (NSTSGGSSNDN;
- SEQ ID NO:
- 62);
- or
- d) SEQ ID NO:90, which corresponds to an AAV3B capsid protein comprising an S268T modification.
6 more elements
A method of altering tissue tropism of an adeno-associated virus (AAV) comprising:
- a) modifying the liver toggle site in an AAV9 capsid protein to comprise AAV3B-VR1 (SQSGASNDN, SEQ ID NO:
- 58), which results in a capsid protein having the amino acid sequence as set forth in SEQ ID NO:87;
- b) modifying the liver toggle site in an AAV80L65 capsid protein to comprise AAV3B-VR1 (SQSGASNDN, SEQ ID NO:
- 58), which results in a capsid protein having the amino acid sequence as set forth in SEQ ID NO:88;
- c) modifying the liver toggle site in an AAV80L65 capsid protein to comprise AAV9-VR1 (NSTSGGSSNDN;
- SEQ ID NO:
- 62), which results in a capsid protein having the amino acid sequence as set forth in SEQ ID NO:89;
- or
- d) modifying the liver toggle site in an AAV3B capsid protein to comprise an S268T modification, which results in a capsid protein having the amino acid sequence as set forth in SEQ ID NO:90.
6 more elements
Dependencies are inferred from the claim text.