Recombinant AAVS having useful transcytosis properties
AssigneeUniversity of Massachusetts
InventorsGuangping Gao, Li Zhong
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Abstract
The disclosure in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the disclosure relates to gene transfer methods using the recombinant adeno-associate viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.
Claims20
A recombinant AAV (rAAV) comprising (i) an AAV capsid protein that is encoded by an isolated nucleic acid comprising a sequence selected from the group consisting of:
- SEQ ID NOs:
- 10-14;
- and (ii) at least one transgene.
A method for treating Canavan disease, the method comprising administering to a subject a therapeutically effective amount of a rAAV to a subject, wherein the rAAV comprises:
- (i) a capsid protein that is encoded by an isolated nucleic acid comprising having a sequence selected from SEQ ID NOs:
- 10 to 14, and
- (ii) at least one transgene, wherein the at least one transgene encodes ASPA;
- and wherein the rAAV infects cells of a target tissue of the subject.
A method for treating cancer, the method comprising administering to a subject a therapeutically effective amount of a rAAV to a subject, wherein the rAAV comprises:
- (i) a capsid protein that is encoded by an isolated nucleic acid comprising having a sequence selected from SEQ ID NOs:
- 10 to 14, and
- (ii) at least one transgene,
- wherein the at least one transgene encodes (a) a tumor suppressor protein or (b) a siRNA, a microRNA, or an antisense nucleotide that inhibits the expression of a gene product associated with cancer;
- and wherein the rAAV infects cells of a target tissue of the subject.
Dependencies are inferred from the claim text.