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US5587308A
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Modified adeno-associated virus vector capable of expression from a novel promoter
Assignee: United States Department of Health and Human Services
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Filing: 1992-06-02
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Granted: 1996-12-24
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Expires: 2012-06-02
Abstract
Abstract Described herein are constructions of recombinant DNA comprising modified adeno-associated virus (AAV) DNA sequences capable of functioning as a eukaryotic expression vector for expressing foreign DNA sequences using a novel transcription promoter comprising the termini of AAV DNA. It is shown that expression of a test reporter gene can be obtained from this vector in mammalian cells. It is further shown that this combination of vector and promoter can be used to introduce and express a human gene and correct a genetic defect in human cells resulting from malfunction of the mutant endogenous gene. Further, the vector can be used to correct the genetic defect by expressing a modified version of the human gene consisting of a fusion of part of the said gene and a synthetic sequence contained in the vector.
Inventors
- 👤 Barrie J. Carter
- 👤 Terence Flotte
- 👤 Sandra Afione
- 👤 Rikki Solow
Bibliographic Details
Application Number
—
Jurisdiction
US
Data Source
google_patents
Last Synchronized
2026-11-08 10:28
CPC Classifications
C; C12; C12N; C12N15/00; C12N15/09; C12N15/63; C12N15/79; C12N15/85; C12N15/86; C07; C07K; C07K14/00; C07K14/435; C07K14/46; C07K14/47; C07K14/4701; C07K14/4712; A; A61; A61K; A61K38/00; A61K48/00; C07K2319/00; C12N2750/00; C12N2750/00011; C12N2750/14011; C12N2750/14111; C12N2750/14141; C12N2750/14143
Claims Analysis
Total Claims: 14
Claim 1
Independent Claim
55 words
An adeno-associated viral vector in an adeno-associated virus particle comprising the inverted terminal repeat sequences of adeno-associated virus and a nucleic acid of a size within the adeno-associated virus packaging limit, wherein the inverted terminal repeat sequences promote expression of the nucleic acid, in the absence of another promoter functionally linked to the nucleic acid.
Claim 2
Dependent Claim
55 words
An adeno-associated viral vector in an adeno-associated virus particle comprising the inverted terminal repeat sequences of adeno-associated virus and a nucleic acid of a size within the adeno-associated virus packaging limit, wherein the inverted terminal repeat sequences promote expression of the nucleic acid, in the absence of another promoter functionally linked to the nucleic acid.
Claim 3
Dependent Claim
12 words
The vector of claim 1, wherein the nucleic acid encodes a polypeptide.
Claim 4
Dependent Claim
10 words
The vector of claim 1 in a pharmaceutically acceptable carrier.
Claim 5
Dependent Claim
12 words
The vector of claim 2, wherein the polypeptide is a functional protein.
Claim 6
Dependent Claim
15 words
The vector of claim 4, wherein the protein is the cystic fibrosis transmembrane conductance regulator.
Claim 7
Dependent Claim
9 words
A host cell containing the vector of claim 5.
Claim 8
Dependent Claim
14 words
The host cell of claim 6, wherein the host cell is an epithelial cell.
Claim 9
Dependent Claim
30 words
A vector comprising a polyA site that can be translationally read in the reverse direction, wherein the poly A site has the nucleotide sequence as shown in SEQ ID NO:13.
Claim 10
Dependent Claim
30 words
A vector comprising a polyA site that can be translationally read in the reverse direction, wherein the poly A site has the nucleotide sequence as shown in SEQ ID NO:13.
Claim 11
Dependent Claim
67 words
An adeno-associated viral vector in an adeno-associated virus particle comprising a. a polyA site that can be translationally read in the reverse direction; b. the inverted terminal repeat sequences of adeno-associated virus; and c. a nucleic acid of a size within the adeno-associated virus packaging limit encoding a polypeptide, wherein the inverted terminal repeat sequences promote expression of the nucleic acid in the absence of another promoter.
Claim 12
Dependent Claim
67 words
An adeno-associated viral vector in an adeno-associated virus particle comprising a. a polyA site that can be translationally read in the reverse direction; b. the inverted terminal repeat sequences of adeno-associated virus; and c. a nucleic acid of a size within the adeno-associated virus packaging limit encoding a polypeptide, wherein the inverted terminal repeat sequences promote expression of the nucleic acid in the absence of another promoter.
Claim 13
Dependent Claim
39 words
A plasmid comprising the inverted terminal repeat sequences of adeno-associated virus and a nucleic acid, wherein the inverted terminal repeat sequences promote expression of the nucleic acid, in the absence of another promoter functionally linked to the nucleic acid.
Claim 14
Dependent Claim
39 words
A plasmid comprising the inverted terminal repeat sequences of adeno-associated virus and a nucleic acid, wherein the inverted terminal repeat sequences promote expression of the nucleic acid, in the absence of another promoter functionally linked to the nucleic acid.
References Cited (Backward Citations)
US4797368A
Cited
USPTO Annuity & Maintenance Windows
| Annuity Stage | Window Opens | Payment Deadline | Surcharge Period Until | Estimated Fee | Status |
|---|---|---|---|---|---|
| 3.5 Years (Year 4) | 1999-12-24 | 2000-06-24 | 2000-12-24 | $2000.00 | Window Closed / Lapsed |
| 7.5 Years (Year 8) | 2003-12-24 | 2004-06-24 | 2004-12-24 | $3760.00 | Window Closed / Lapsed |
| 11.5 Years (Year 12) | 2007-12-24 | 2008-06-24 | 2008-12-24 | $7700.00 | Window Closed / Lapsed |