Aav vectors for retinal and cns gene therapy
US-2017096683-A1 · Apr 6, 2017 · US
Methods and compositions for modifying assembly-activating protein (AAP)-dependence of viruses
US11034732B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-11034732-B2 |
| Application number | US-201916409317-A |
| Country | US |
| Kind code | B2 |
| Filing date | May 10, 2019 |
| Priority date | May 10, 2017 |
| Publication date | Jun 15, 2021 |
| Grant date | Jun 15, 2021 |
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- Title
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Abstract
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Methods and compositions are provided that can be used to modify the assembly activating protein (AAP)-dependence of an adeno-associated virus (AAV).
First claim
Opening claim text (preview).
What is claimed is: 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.
Assignees
Inventors
Classifications
viral genome or elements thereof as genetic vector · CPC title
- C07K14/005Primary
from viruses · CPC title
Viral vectors · CPC title
New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes · CPC title
- C07K14/015Primary
Parvoviridae, e.g. feline panleukopenia virus, human parvovirus · CPC title
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Frequently asked questions
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- What does patent US11034732B2 cover?
- Methods and compositions are provided that can be used to modify the assembly activating protein (AAP)-dependence of an adeno-associated virus (AAV).
- Who is the assignee on this patent?
- Massachusetts Eye & Ear Infirmary, Schepens Eye Res Inst
- What technology area does this patent fall under?
- Primary CPC classification C07K14/005. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Jun 15 2021 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 3 related publications on this page (citations in our corpus or others sharing the same primary CPC).