Patent US531282A successfully refreshed from Google Patents.
Patent US5652224A successfully refreshed from Google Patents.
US5652224A
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Methods and compositions for gene therapy for the treatment of defects in lipoprotein metabolism
Assignee: University of Pennsylvania
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Filing: 1995-02-24
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Granted: 1997-07-29
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Expires: 2015-02-24
Abstract
Abstract The invention provides a recombinant viral vector comprising the DNA of, or corresponding to, at least a portion of the genome of an adenovirus, which portion is capable of infecting a hepatic cell; and a human VLDL receptor gene operatively linked to regulatory sequences directing its expression. The vector is capable of expressing the normal VLDL receptor gene product in hepatic cells in vivo or in vitro. This viral vector is useful in the treatment of metabolic disorders caused by the accumulation of LDL in plasma, such as familial hypercholesterolemia or familial combined hyperlipidemia.
Inventors
- 👤 James M. Wilson
- 👤 Karen Kozarsky
- 👤 Jerome Strauss, III
Bibliographic Details
Application Number
—
Jurisdiction
US
Data Source
google_patents
Last Synchronized
2026-11-03 06:50
CPC Classifications
C; C12; C12N; C12N15/00; C12N15/09; C12N15/63; C12N15/79; C12N15/85; C12N15/86; A; A61; A61P; A61P3/00; A61P3/06; C07; C07K; C07K14/00; C07K14/435; C07K14/705; A61K; A61K38/00; A61K48/00; C12N2710/00; C12N2710/00011; C12N2710/10011; C12N2710/10311; C12N2710/10341; C12N2710/10344; C12N2799/00; C12N2799/02; C12N2799/021; C12N2799/022
Claims Analysis
Total Claims: 19
Claim 1
Independent Claim
24 words
A recombinant adenoviral vector comprising a human VLDL receptor gene operatively linked to regulatory sequences directing expression of said receptor gene in a hepatocyte.
Claim 2
Dependent Claim
24 words
A recombinant adenoviral vector comprising a human VLDL receptor gene operatively linked to regulatory sequences directing expression of said receptor gene in a hepatocyte.
Claim 3
Dependent Claim
19 words
The vector according to claim 1 further comprising adenovirus 5' and 3' cis-elements necessary for replication and virion encapsidation.
Claim 4
Dependent Claim
19 words
The vector according to claim 1 further comprising a deletion in all or a portion of the E1 gene.
Claim 5
Dependent Claim
19 words
The vector according to claim 1 further comprising a deletion in all or a portion of the E3 gene.
Claim 6
Independent Claim
44 words
A method for delivering a VLDL receptor gene into a hepatocyte comprising introducing into said hepatocyte an effective amount of a recombinant adenoviral vector comprising a human VLDL receptor gene operatively linked to regulatory sequences directing expression of the VLDL receptor in said hepatocyte.
Claim 7
Independent Claim
44 words
A method for delivering a VLDL receptor gene into a hepatocyte comprising introducing into said hepatocyte an effective amount of a recombinant adenoviral vector comprising a human VLDL receptor gene operatively linked to regulatory sequences directing expression of the VLDL receptor in said hepatocyte.
Claim 8
Dependent Claim
40 words
A mammalian hepatocyte, which expresses a human VLDL receptor introduced therein through transduction of said hepatocyte by an adenoviral vector comprising a human VLDL receptor gene operatively linked to regulatory sequences directing expression of said receptor gene in said hepatocyte.
Claim 9
Dependent Claim
40 words
A mammalian hepatocyte, which expresses a human VLDL receptor introduced therein through transduction of said hepatocyte by an adenoviral vector comprising a human VLDL receptor gene operatively linked to regulatory sequences directing expression of said receptor gene in said hepatocyte.
Claim 10
Independent Claim
53 words
A method for treating a patient having a disorder characterized by an elevated concentration of LDL in plasma comprising administering into the bloodstream of said patient an effective amount of a recombinant adenoviral vector comprising: a human VLDL receptor gene operatively linked to regulatory sequences directing expression of the VLDL receptor in hepatocytes.
Claim 11
Independent Claim
53 words
A method for treating a patient having a disorder characterized by an elevated concentration of LDL in plasma comprising administering into the bloodstream of said patient an effective amount of a recombinant adenoviral vector comprising: a human VLDL receptor gene operatively linked to regulatory sequences directing expression of the VLDL receptor in hepatocytes.
Claim 12
Dependent Claim
16 words
The method according to claim 7 wherein said disorder is familial hypercholesterolemia or familial combined hyperlipidemia.
Claim 13
Independent Claim
50 words
A method for decreasing the levels of VLDL and LDL in the plasma of a patient, comprising administering into the bloodstream of said patient an effective amount of an adenoviral vector comprising a human VLDL receptor gene operatively linked to regulatory sequences directing expression of the VLDL receptor in hepatocytes.
Claim 14
Independent Claim
50 words
A method for decreasing the levels of VLDL and LDL in the plasma of a patient, comprising administering into the bloodstream of said patient an effective amount of an adenoviral vector comprising a human VLDL receptor gene operatively linked to regulatory sequences directing expression of the VLDL receptor in hepatocytes.
Claim 15
Dependent Claim
34 words
A pharmaceutical composition comprising a pharmaceutically acceptable carrier and an adenoviral vector, said vector comprising a human VLDL receptor gene operatively linked to regulatory sequences directing expression of said receptor gene in a hepatocyte.
Claim 16
Dependent Claim
34 words
A pharmaceutical composition comprising a pharmaceutically acceptable carrier and an adenoviral vector, said vector comprising a human VLDL receptor gene operatively linked to regulatory sequences directing expression of said receptor gene in a hepatocyte.
Claim 17
Dependent Claim
22 words
The composition according to claim 10 wherein said adenoviral vector comprises adenovirus 5' and 3' cis-elements necessary for replication and virion encapsidation.
Claim 18
Dependent Claim
22 words
The composition according to claim 10 wherein said adenoviral vector comprises a deletion in all or a portion of the E1 gene.
Claim 19
Dependent Claim
22 words
The composition according to claim 10 wherein said adenoviral vector comprises a deletion in all or a portion of the E3 gene.
References Cited (Backward Citations)
US4658019A
Cited
US5017691A
Cited
US5166320A
Cited
WO1990005147A1
Cited
US5240846A
Cited
WO1991018088A1
Cited
EP0501233A1
Cited
WO1993000431A1
Cited
EP0555880A2
Cited
WO1994010322A1
Cited
WO1994012649A2
Cited
WO1994017832A1
Cited
WO1995006743A2
Cited
WO1995013374A2
Cited
USPTO Annuity & Maintenance Windows
| Annuity Stage | Window Opens | Payment Deadline | Surcharge Period Until | Estimated Fee | Status |
|---|---|---|---|---|---|
| 3.5 Years (Year 4) | 2000-07-28 | 2001-01-27 | 2001-07-29 | $2000.00 | Window Closed / Lapsed |
| 7.5 Years (Year 8) | 2004-07-28 | 2005-01-27 | 2005-07-29 | $3760.00 | Window Closed / Lapsed |
| 11.5 Years (Year 12) | 2008-07-28 | 2009-01-27 | 2009-07-29 | $7700.00 | Window Closed / Lapsed |