IP Library › Granted Patent US 11,034,732
Granted Patent B2
US 11,034,732 · App. 16/409,317 · Granted Jun 15, 2021

Methods and compositions for modifying assembly-activating protein (AAP)-dependence of viruses

Inventors: Luk H. Vandenberghe (Weston, MA); Anna Claire Maurer (Boston, MA)
Assignees: Massachusetts Eye and Ear Infirmary; The Schepens Eye Research Institute, Inc.
C07K14/015C12N15/86C12N2750/14122C12N2750/14143C12N2750/14152
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Quick Facts
Patent No.
US 11,034,732
App. No.
16/409,317
Granted
Jun 15, 2021
Kind
B2
Abstract

Methods and compositions are provided that can be used to modify the assembly activating protein (AAP)-dependence of an adeno-associated virus (AAV).

Claims (14)

1. An adeno-associated virus (AAV) capsid polypeptide comprising an amino acid sequence having at least 99% sequence identity to the amino acid sequence of SEQ ID NO: 3.

2. The AAV capsid polypeptide of claim 1 , having the amino acid sequence of SEQ ID NO:3.

3. The AAV capsid polypeptide of claim 1 , encoded by the nucleic acid sequence of SEQ ID NO: 4.

4. A virus particle comprising the adeno-associated virus (AAV) capsid polypeptide of claim 1 .

5. The virus particle of claim 4 , further comprising a transgene.

6. A nucleic acid molecule comprising a nucleic acid sequence encoding the adeno-associated virus (AAV) capsid polypeptide of claim 1 .

7. The nucleic acid molecule of claim 6 , having the nucleic acid sequence of SEQ ID NO: 4.

8. The nucleic acid molecule of claim 6 , encoding the amino acid sequence of SEQ ID NO: 3.

9. A vector comprising the nucleic acid molecule of claim 6 .

10. A host cell comprising the nucleic acid molecule of claim 6 .

11. The host cell of claim 10 , wherein the host cell is a packaging cell.

12. A packaging cell comprising a nucleic acid molecule encoding the adeno-associated virus (AAV) capsid polypeptide of claim 1 .

13. The packaging cell of claim 12 , wherein the packaging cell lacks an assembly activating protein (AAP).

14. An AAV capsid polypeptide comprising the amino acid sequence of SEQ ID NO:1 comprising amino acid substitutions K163T, A2065, K478R, L481V, V520M, T528S, L586N, A592Q, P593A, N599H, S600N, and A603V.

Assignments (2)
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 20, 2019
From: VANDENBERGHE, LUK H.
To: THE SCHEPENS EYE RESEARCH INSTITUTE, INC.; MASSACHUSETTS EYE AND EAR INFIRMARY
Reel/Frame 049536/0929 →
ASSIGNMENT OF ASSIGNOR'S INTEREST Recorded Jun 20, 2019
From: MAURER, ANNA CLAIRE
To: THE SCHEPENS EYE RESEARCH INSTITUTE, INC.
Reel/Frame 049537/0021 →
Continuity (4)
Continuation PCTUS2018032166 · May 10, 2018
Provisional Application 62669901 · May 10, 2018
Provisional Application 62504318 · May 10, 2017
Related Publication 20190330278A1 · Oct 31, 2019
Cited By (2)
US 12,227,543 US 12,359,174