US6180613B1
Expired / Lapsed
US
⏱️ 0.0 years remaining
AAV-mediated delivery of DNA to cells of the nervous system
Assignee: Yale University
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Filing: 1995-06-06
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Granted: 2001-01-30
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Expires: 2015-06-06
Abstract
Abstract The invention relates to a method of delivering exogenous DNA to a target cell of the mammalian central nervous system using an adeno-associated virus (AAV)-derived vector. Also included in the invention are the AAV-derived vectors containing exogenous DNA which encodes a protein or proteins which treat nervous system disease, and a method of treating such disease.
Inventors
- 👤 Michael G. Kaplitt
- 👤 Matthew J. During
Bibliographic Details
Application Number
—
Jurisdiction
US
Data Source
google_patents
Last Synchronized
2026-11-07 14:10
CPC Classifications
C; C12; C12N; C12N15/00; C12N15/09; C12N15/63; C12N15/79; C12N15/85; C12N15/86; A; A61; A61P; A61P21/00; A61P25/00; A61P25/08; A61P25/14; A61P25/16; A61P25/28; A61P35/00; A61P39/00; A61P39/06; A61P43/00; A61P5/00; A61P9/00; C07; C07K; C07K14/00; C07K14/435; C07K14/475; C07K14/48; C12N9/00; C12N9/0004; C12N9/0071; C12N9/0073; C12N9/88; C12Y; C12Y114/00; C12Y114/16; C12Y114/16002; C12Y401/00; C12Y401/01; C12Y401/01028; A61K; A61K38/00; A61K48/00; C12N2750/00; C12N2750/00011; C12N2750/14011; C12N2750/14111; C12N2750/14141; C12N2750/14143; C12N2840/00; C12N2840/20; C12N2840/203
Claims Analysis
Total Claims: 17
Claim 1
Independent Claim
108 words
A method for ameliorating a symptom of a central nervous system disorder in a mammal, the method comprising, direct administration of an adeno-associated virus-derived vector to a target cell in the brain of the mammal, said vector comprising a DNA sequence, wherein said DNA sequence is exogenous to an adeno-associated virus and comprises a sequence encoding a therapeutic protein in operable linkage with a promoter sequence, wherein said adeno-associated virus-derived vector is free of both wild-type and helper virus, and wherein the exogenous DNA sequence is expressed in the target cell for greater than 3 months such that the symptom of the central nervous system disorder is ameliorated.
Claim 2
Independent Claim
108 words
A method for ameliorating a symptom of a central nervous system disorder in a mammal, the method comprising, direct administration of an adeno-associated virus-derived vector to a target cell in the brain of the mammal, said vector comprising a DNA sequence, wherein said DNA sequence is exogenous to an adeno-associated virus and comprises a sequence encoding a therapeutic protein in operable linkage with a promoter sequence, wherein said adeno-associated virus-derived vector is free of both wild-type and helper virus, and wherein the exogenous DNA sequence is expressed in the target cell for greater than 3 months such that the symptom of the central nervous system disorder is ameliorated.
Claim 3
Dependent Claim
23 words
The method of claim 1 , wherein the exogenous DNA sequence is expressed in said target cell either constitutively or under regulatable conditions.
Claim 4
Dependent Claim
27 words
The method of claim 2 , wherein the exogenous DNA sequence encodes a protein selected from the group consisting of tyrosine hydroxylase and aromatic amino acid decarboxylase.
Claim 5
Dependent Claim
20 words
The method of claim 2 , wherein the exogenous DNA sequence encodes a specific neurotrophic factor for mesencephalic dopaminergic neurons.
Claim 6
Dependent Claim
35 words
The method of claim 4 , wherein the specific neurotrophic factor for mesencephalic dopaminergic neurons is selected from the group consisting of glial-derived neurotrophic factor (GDNF), brain-derived neurotrophic factor (BDNF), and nerve growth factor (NGF).
Claim 7
Dependent Claim
19 words
The method of claim 1 , wherein all adeno-associated viral genes of the vector have been deleted or inactivated.
Claim 8
Dependent Claim
18 words
The method of claim 1 , wherein the vector comprises only the inverted terminal repeats of adeno-associated virus.
Claim 9
Dependent Claim
16 words
The method of claim 1 , wherein said promoter sequence is a central nervous system-specific promoter.
Claim 10
Dependent Claim
14 words
The method of claim 8 , wherein said promoter sequence is a neuron-specific promoter.
Claim 11
Dependent Claim
14 words
The method of claim 8 , wherein said promoter sequence is a glia-specific promoter.
Claim 12
Dependent Claim
29 words
The method of claim 1 , wherein the target cell is in a region of the brain selected from the group consisting of cerebellum, striatum, hippocampus, and substantia nigra.
Claim 13
Dependent Claim
20 words
The method of claim 1 , wherein the target cell is a primate target cell or a rat target cell.
Claim 14
Dependent Claim
16 words
The method of claim 12 , wherein the primate target cell is a human target cell.
Claim 15
Dependent Claim
13 words
The method of claim 1 , wherein direct administration is by stereotaxic injection.
Claim 16
Dependent Claim
39 words
An adeno-associated virus-derived vector comprising only the replication and packaging signals of adeno-associated virus, and further comprising a DNA molecule comprising a DNA sequence encoding tyrosine hydroxylase, a DNA sequence encoding aromatic amino acid decarboxylase, and a promoter sequence.
Claim 17
Dependent Claim
39 words
An adeno-associated virus-derived vector comprising only the replication and packaging signals of adeno-associated virus, and further comprising a DNA molecule comprising a DNA sequence encoding tyrosine hydroxylase, a DNA sequence encoding aromatic amino acid decarboxylase, and a promoter sequence.
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USPTO Annuity & Maintenance Windows
| Annuity Stage | Window Opens | Payment Deadline | Surcharge Period Until | Estimated Fee | Status |
|---|---|---|---|---|---|
| 3.5 Years (Year 4) | 2004-01-30 | 2004-07-31 | 2005-01-30 | $2000.00 | Window Closed / Lapsed |
| 7.5 Years (Year 8) | 2008-01-30 | 2008-07-31 | 2009-01-30 | $3760.00 | Window Closed / Lapsed |
| 11.5 Years (Year 12) | 2012-01-30 | 2012-07-31 | 2013-01-30 | $7700.00 | Window Closed / Lapsed |