Granted Patent
B2
US 12,291,719 · App. 17/237,095 · Granted May 6, 2025
Heterologous targeting peptide grafted AAVs
Assignee:
University of Massachusetts
C12N15/8645A61K48/0025A61K48/0058C07K7/00C07K7/04C07K14/005C07K14/775C07K16/00C12N15/79C12N15/85C12N15/86C07K14/00C07K14/075C07K19/00C07K2319/01C07K2319/30C07K2319/33C12N2310/141C12N2750/14145C12N2810/85
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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-associated viruses. In some aspects, the disclosure relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.
Claims (23)
1. A method for delivering a transgene to a subject comprising:
administering a rAAV to a subject, wherein the rAAV comprises:
(i) a capsid protein having an N-terminally grafted heterologous targeting peptide, wherein:
(a) the capsid protein is a VP2 capsid protein that is not of an AAV2 serotype; and
(b) the targeting peptide is a CNS-targeting polypeptide consisting of the amino acid sequence set forth in SEQ ID NO: 5, that is inserted between the first and second amino acid residues of the VP2 capsid protein, and
(ii) a recombinant AAV vector comprising a transgene,
wherein the rAAV infects cells of a target tissue of the subject.
2. The method of claim 1 , wherein the transgene encodes a protein.
3. The method of claim 2 , wherein the protein encoded by the transgene is an immunoglobulin heavy chain or light chain or fragment thereof.
4. The method of claim 1 , wherein the transgene encodes a small interfering nucleic acid.
5. The method of claim 4 , wherein the small interfering nucleic acid is a miRNA.
6. The method of claim 4 , wherein the small interfering nucleic acid is a miRNA sponge or tough decoy RNA (TuD RNA) that inhibits the activity of at least one miRNA in the subject or animal.
7. The method of claim 1 , wherein the transgene expresses a transcript that comprises at least one binding site for a miRNA, wherein the miRNA inhibits activity of the transgene, in a tissue other than the target tissue, by hybridizing to the binding site.
8. The method of claim 1 , wherein the at least one transgene encodes a gene product that mediates genome editing.
9. The method of claim 1 , wherein the transgene comprises a tissue specific promoter or inducible promoter.
10. The method of claim 9 , wherein the tissue specific promoter is a liver-specific thyroxin binding globulin (TBG) promoter, an insulin promoter, a glucagon promoter, a somatostatin promoter, a pancreatic polypeptide (PPY) promoter, a synapsin-1 (Syn) promoter, a creatine kinase (MCK) promoter, a mammalian desmin (DES) promoter, a a-myosin heavy chain (a-MHC) promoter, or a cardiac Troponin T (cTnT) promoter.
11. The method of claim 1 , wherein the rAAV is administered intravenously, intravascularly, transdermally, intraocularly, intrathecally, orally, intramuscularly, subcutaneously, intranasally, or by inhalation.
12. The method of claim 1 , wherein the subject is selected from a mouse, a rat, a rabbit, a dog, a cat, a sheep, a pig, and a non-human primate.
13. The method of claim 1 , wherein the subject is a human.
14. The method of claim 1 , wherein the rAAV further comprises a linker conjugated to the C-terminus of the N-terminally grafted heterologous targeting peptide.
15. The method of claim 14 , wherein the linker comprise at least one polypeptide repeat, each repeat comprising at least two glycine residues.
16. The method of claim 15 , wherein the linker is of the formula [GGGGS SEQ ID NO: 28]n, wherein n is an integer in a range of 1 to 4.
17. The method of claim 1 , wherein the capsid protein comprises an AAV9 capsid protein.
Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Jul 23, 2021
From: ESTEVES, MIGUEL SENA; CHOUDHURY, SOURAV ROY
To: UNIVERSITY OF MASSACHUSETTS
Reel/Frame 056962/0067 →
Continuity (4)
Continuation
15516582
Provisional Application
62059738
· Oct 3, 2014
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