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US 2026/0295079 A1Published application
Modified aav vectors for gene therapy
AssigneeThe Trustees of Indiana University
InventorsWeidong XIAO, Anh Kim LAM
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Abstract
Disclosed herein are compositions comprising chemically modified adeno-associated viruses (AAV) engineered by chemical modification of capsid proteins, whether wildtype or engineered, and methods of using such modified AAVs for therapeutic use wherein the modified AAVs are re-directed to specific organs/tissues relative to the unmodified AAV.
Claims20
A modified adeno-associated virus (AAV) comprising:
- (i) an outer shell comprising capsid proteins and at least one chemical moiety covalently bound to at least one capsid protein, the at least one capsid protein selected from VP1, VP2, or VP3, and
- (ii) a single strand of DNA (ssDNA), wherein the ssDNA is recombinant DNA (rDNA), wherein the at least one chemical moiety is covalently bound to a lysine residue present in the at least one capsid protein, and
- wherein the at least on chemical moiety alters the tropism of the modified AAV relative to an unmodified AAV.
A method of altering the tropism of an adeno-associated virus (AAV) upon administration to a subject comprising:
- chemically modifying a capsid protein of the AAV by the covalent attachment of a chemical moiety to form a modified AAV, wherein the chemical moiety alters the tropism of the modified AAV compared to an unmodified AAV.
Dependencies are inferred from the claim text.