IP Library › Granted Patent US 10,689,420
Granted Patent B2
US 10,689,420 · App. 16/019,636 · Granted Jun 23, 2020

AAV's and uses thereof

Inventors: Guangping Gao (Westborough, MA); Terence Flotte (Holden, MA); Jun Xie (Shrewsbury, MA)
Assignee: University of Massachusetts
C07K14/005A61K31/7088A61K35/76C12N7/00C12N15/86A61K48/00C12N2750/14122C12N2750/14143C12N2750/14145
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Quick Facts
Patent No.
US 10,689,420
App. No.
16/019,636
Granted
Jun 23, 2020
Kind
B2
Abstract

The invention in some aspects relates to recombinant adeno-associated viruses having distinct tissue targeting capabilities. In some aspects, the invention relates to gene transfer methods using the recombinant adeno-associate viruses. In some aspects, the invention relates to isolated AAV capsid proteins and isolated nucleic acids encoding the same.

Claims (18)

1. A recombinant adeno-associated virus (rAAV) comprising a nucleic acid encoding a heterologous transgene and an AAV capsid, wherein the AAV capsid comprises a protein comprising an amino acid sequence selected from SEQ ID NOs.: 96, 98, 100, and 111.

2. The rAAV of claim 1 , wherein the heterologous transgene comprises a tissue-specific promoter operably linked to a coding sequence.

3. The rAAV of claim 2 , wherein the coding sequence encodes a therapeutic protein.

4. The rAAV of claim 3 , wherein the therapeutic protein is aromatic amino acid decarboxylase (AADC).

5. A composition comprising the rAAV of claim 1 .

6. The composition of claim 5 , further comprising a pharmaceutically acceptable carrier.

7. The rAAV of claim 1 , wherein the AAV capsid protein comprises the amino acid sequence set forth in SEQ ID NO.: 96.

8. A composition comprising the rAAV of claim 7 and a pharmaceutically acceptable carrier.

9. The rAAV of claim 1 , wherein the AAV capsid protein comprises the amino acid sequence set forth in SEQ ID NO.: 98.

10. A composition comprising the rAAV of claim 9 and a pharmaceutically acceptable carrier.

11. The rAAV of claim 1 , wherein the AAV capsid protein comprises the amino acid sequence set forth in SEQ ID NO.: 100.

12. A composition comprising the rAAV of claim 11 and a pharmaceutically acceptable carrier.

13. The rAAV of claim 1 , wherein the AAV capsid protein comprises the amino acid sequence set forth in SEQ ID NO.: 111.

14. A composition comprising the rAAV of claim 13 and a pharmaceutically acceptable carrier.

15. The rAAV of claim 3 , wherein the AAV capsid protein comprises the amino acid sequence set forth in SEQ ID NO.: 96.

16. The rAAV of claim 3 , wherein the AAV capsid protein comprises the amino acid sequence set forth in SEQ ID NO.: 98.

17. The rAAV of claim 3 , wherein the AAV capsid protein comprises the amino acid sequence set forth in SEQ ID NO.: 100.

18. The rAAV of claim 3 , wherein the AAV capsid protein comprises the amino acid sequence set forth in SEQ ID NO.: 111.

Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Dec 19, 2018
From: GAO, GUANGPING; FLOTTE, TERENCE; XIE, JUN
To: UNIVERSITY OF MASSACHUSETTS
Reel/Frame 047812/0748 →
Continuity (5)
Continuation 15007559 · Jan 27, 2016
Continuation 14246560 · Apr 7, 2014
Continuation 13322164
Provisional Application 61182084 · May 28, 2009
Related Publication 20180362592A1 · Dec 20, 2018