US5240846A
Expired / Lapsed
US
⏱️ 0.0 years remaining
Gene therapy vector for cystic fibrosis
Assignee: Hospital for Sick Children
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Filing: 1990-09-18
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Granted: 1993-08-31
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Expires: 2010-09-18
Abstract
Abstract The present invention comprises gene therapy for treating cystic fibrosis(CF). Delivery and expression of a single copy of a normal CFTR gene leads to stable correction of the Cl channel regulation defect present in CF epithelial cells. The present invention includes recombinant viral and plasmid vectors, alternative CFTR gene delivery strategies, and transduced CF cells and cell lines carrying a recombinant gene for functional CFTR. CF epithelial complementation through transduction of the present invention also provides an assay for determining the validity of other putative CF mutations.
Inventors
- 👤 Francis S. Collins
- 👤 James M. Wilson
Bibliographic Details
Application Number
—
Jurisdiction
US
Data Source
google_patents
Last Synchronized
2026-11-03 00:02
CPC Classifications
B; B82; B82Y; B82Y5/00; C; C07; C07K; C07K14/00; C07K14/435; C07K14/46; C07K14/47; C07K14/4701; C07K14/4712; C07K16/00; C07K16/18; C07K16/28; C12; C12N; C12N15/00; C12N15/09; C12N15/63; C12N15/79; C12N15/85; C12N15/8509; A; A01; A01K; A01K2217/00; A01K2217/05; A01K2267/00; A01K2267/03; A01K2267/0306; A61; A61K; A61K38/00; A61K48/00
Claims Analysis
Total Claims: 18
Claim 1
Independent Claim
76 words
A recombinant viral vector for treating a defect in the gene for cystic fibrosis transmembrane regulator in a target cell, the vector comprising: a) the DNA of or corresponding to at least a portion of the genome of a virus which portion is capable of infecting the target cells; and b) a normal cystic fibrosis transmembrane regulator gene operatively linked to the DNA and capable of expression in the target cell in vivo or in vitro.
Claim 2
Dependent Claim
76 words
A recombinant viral vector for treating a defect in the gene for cystic fibrosis transmembrane regulator in a target cell, the vector comprising: a) the DNA of or corresponding to at least a portion of the genome of a virus which portion is capable of infecting the target cells; and b) a normal cystic fibrosis transmembrane regulator gene operatively linked to the DNA and capable of expression in the target cell in vivo or in vitro.
Claim 3
Dependent Claim
12 words
The recombinant vector of claim 1, wherein the virus is a retrovirus.
Claim 4
Dependent Claim
12 words
The recombinant vector of claim 2, wherein the retroviral genome is replication-defective.
Claim 5
Dependent Claim
9 words
The recombinant vector of claim 2, further comprising pLJ.
Claim 6
Dependent Claim
17 words
The recombinant vector of claim 4, wherein the vector provirus is substantially as shown in FIG. 1A.
Claim 7
Dependent Claim
14 words
The recombinant vector of claim 2, wherein the defect being treated causes cystic fibrosis.
Claim 8
Dependent Claim
14 words
The recombinant vector of claim 1, wherein the target cell is an epithelial cell.
Claim 9
Dependent Claim
12 words
The recombinant vector of claim 6, wherein the epithelial cell is pancreatic.
Claim 10
Dependent Claim
15 words
The recombinant vector of claim 6, wherein the epithelial cell is an airway epithelial cell.
Claim 11
Dependent Claim
26 words
The recombinant vector of claim 6, wherein the epithelial cell is a cell selected from the group consisting of sweat gland, intestinal, liver and kidney cells.
Claim 12
Dependent Claim
9 words
The recombinant vector of claim 7, wherein comprising pLJ.
Claim 13
Dependent Claim
9 words
The recombinant vector of claim 8, further comprising pLJ.
Claim 14
Dependent Claim
24 words
The recombinant vector of claim 4, wherein the normal cystic fibrosis transmembrane regulation gene includes a silent mutation which stabilizes expression of the gene.
Claim 15
Dependent Claim
17 words
A CF cell which expresses a normal cystic fibrosis transmembrane regulator gene introduced therein through retroviral transduction.
Claim 16
Dependent Claim
17 words
A CF cell which expresses a normal cystic fibrosis transmembrane regulator gene introduced therein through retroviral transduction.
Claim 17
Dependent Claim
17 words
The cell of claim 14, wherein the cell is derived from a pLJ-cystic fibrosis transmembrane regulator clone.
Claim 18
Dependent Claim
54 words
The recombinant vector of claim 13, wherein the silent mutation comprises the presence of cytosine at nucleotide position 930 of the cystic fibrosis transmembrane regulator cDNA sequence, guanine at nucleotide position 933 of the cystic fibrosis transmembrane regulator cDNA sequence, and cytosine at nucleotide position 936 of the cystic fibrosis transmembrane regulator cDNA 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) | 1996-08-30 | 1997-03-01 | 1997-08-31 | $2000.00 | Window Closed / Lapsed |
| 7.5 Years (Year 8) | 2000-08-30 | 2001-03-01 | 2001-08-31 | $3760.00 | Window Closed / Lapsed |
| 11.5 Years (Year 12) | 2004-08-30 | 2005-03-01 | 2005-08-31 | $7700.00 | Window Closed / Lapsed |