Adenovirus vectors for gene therapy

Details Adenovirus vectors for gene therapy Adenovirus vectors for gene therapy

1993-06-07

1996-12-17

Stone, Jacqueline M.

Drug, bio-affecting and body treating compositions

Designated organic active ingredient containing

Carbohydrate doai

4353201, 4351723, 4352402, A61K 4800, C12N 516, C12N 1563

Patent

active

055853625

ABSTRACT:
The present invention comprises an improved adenovirus vector and methods for making and using such vectors. The adenovirus vectors of the present invention retain at least a portion of the adenoviral E3 region, carry a deletion of at least a portion of the adenoviral E1 region. Vectors of the present invention preferably also include an additional deletion to accommodate a transgene and/or other mutations which result in reduced expression or over-expression of adenoviral protein and/or reduced viral replication. The vectors of the present invention further include a transgene operatively-linked thereto. By reducing or eliminating viral replication and viral protein expression, the immune response of the infected host to the virus and viral protein is decreased and persistence of transgene expression can be increased. The adenovirus vectors of the present invention are thus particularly useful in gene transfer and therapy.

REFERENCES:
patent: 4844893 (1989-07-01), Honsik et al.
patent: 4847201 (1989-07-01), Kaswasaki et al.
patent: 4853331 (1989-08-01), Herrnstadt et al.
patent: 4861719 (1989-08-01), Miller
patent: 4861859 (1989-08-01), Ju
patent: 4868116 (1989-09-01), Morgan et al.
patent: 4980286 (1990-12-01), Morgan et al.
patent: 5240846 (1993-08-01), Collins et al.
Boat et al., "Human Respiratory Tract Secretions", Arch. Biochem. Biophys. 177:95-104 (1976).
Boucher et al., "Na.sup.+ Transport In Cystic Fibrosis Respiratory Epithelia", J. Clin. Invest. 78:1245-1252 (1986).
Cheng et al., "Increased Sulfation Of Glycoconjugates By Cultured Nasal Epithelial Cells From Patients With Cystic Fibrosis", J. Clin. Invest. 84:68-72 (1989).
Cliff et al., "Separate Cl Conductances Activated By cAMP and Ca.sup.2+ in Cl.sup.- -Secreting Epithelial Cells", PNAS (USA)87:4956-4960 (1990).
Collie et al., "Culture Of Sweat Gland Epithelial Cells From Normal Individuals And Patients With Cystic Fibrosis", In Vitro Cell &Devel. Biol. 21:597-602 (1985).
Collins et al., "Construction Of A General Human Chromosome Jumping Library, With Application To Cystic Fibrosis", Science 235:1046-1049 (1987).
Cutting et al., "A Cluster Of Cystic Fibrosis Mutations In The First Nucleotide-Binding Fold Of The Cystic Fibrosis Conductance Regulation Protein", Nature 346:366-368 (1990).
Dean et al., "Multiple Mutations In Highly Conserved Residues Are Found In Mildly Affected Cystic Fibrosis Patients", Cell 61:863-870 (1990).
Drumm et al., "Physical Mapping Of The Cystic Fibrosis Region By Pulsed-Field Gel Electrophoresis", Genomics 2:346-354 (1988).
Fienberg et al., "A Technique For Radiolabeling DNA Restriction Endonuclease Fragments To High Specific Activity", Anal. Biochem. 132:6-13 (1983).
Frizzell et al., "Cystic Fibrosis: A Disease Of Ion Channels?", Trends Neurosci 10:190-193 (1987).
Frizzell et al., "Altered Regulation Of Airway Epithelial Cell Chloride Channels In Cystic Fibrosis", Science 233:558-560 (1986).
Green et al., "Chromosomal Region Of The Cystic Fibrosis Gene In The Yeast Artificial Chromosomes: A Model For Human Genome Mapping", Science 250:94-98 (1990).
Harris et al., "Establishment Of A Tissue Culture System For Epithelial Cells Desired From Human Pancreas: A Model For The Study Of Cystic Fibrosis", Cell 87:695-703 (1987).
Hyde et al., "Structural Model For ATP-Binding Proteins Associated With Cystic Fibrosis, Multidrug Resistance And Bacterial Transport", Nature 346:362-365 (1990).
Kerem et al., "Identification Of The Cystic Fibrosis Gene: Genetic Analysis", Science 245:1073-1080 (1989).
Kerem et al., "Identification of Mutations in Regions Corresponding to the Two Putative Nucleotide (ATP)-Binding Folds Of The Cystic Fibrosis Gene", PNAS (USA)87:8447-8451 (1990).
Korman et al., "Expression Of Human Class II Major Histocompatibility Complex Antigens Using Retrovirus Vectors", PNAS(USA)85:2150-2154 (1987).
Li et al., "Cyclic AMP-Dependent Protein Kinase Opens Chloride Channels In Normal But Not Cystic Fibrosis Airway Epithelium", Nature 331:358-360 (1988).
Quinton et al., "Cystic Fibrosis: A Disease In Electrolyte Transport", FASEB J. 4:2709-2717 (1990).
Riordan et al., "Identification Of The Cystic Fibrosis Gene: Cloning And Characterization Of Complementary DNA", Science 245:1066-1073 (1989).
Riordan et al., Genetics And Epithelial Cell Dysfunction In Cystic Fibrosis, 59-71 (1987).
Rommens et al., "Identification Of The Cystic Fibrosis Gene: Chromosome Walking And Jumping", Science 245:1059-1065 (1989).
Sanbrook et al., "Oligonucleotide-Mediated Mutagenesis", Molecular Cloning, A Laboratory Manual 2nd Ed., Cold Spring Harbor Press, 15.51-15.80 (1989).
Sato et al., "Defective Beta Adrenergic Response Of Cystic Fibrosis Sweat Glands In Vivo And In Vitro", J. Clin. Invest. 73:1763-1771 (1984).
Schoumacher et al., "A Cystic Fibrosis Pancreatic Adenocarcinoma Cell Line", PNAS (USA)87:4012-4016 (1990).
Smith et al., "In Vitro Mutagenesis", Annu. Re. Genet. 19:423-462 (1985).
Stutts et al., "Chloride Uptake Into Cultured Airway Epithelial Cells From Cystic Fibrosis Patients And Normal Individuals", PNAS (USA)82:6677-6681 (1985).
Taussig et al., "Cystic Fibrosis: An Overview", In Cystic Fibrosis, L. M. Taussig, ed. New York: Thieme-Stralton 1-9 (1984).
Tsui et al., "Cystic Fibrosis Locus Defined By A GEnetically Linked Polymorphic DNA Marker", Science 230:1054-1057 (1985).
Venglarik et al., "A Simple Assay For Agonist-Regulated Cl and K Conductances In Salt-Secreting Epithelial Cells", Am. J. Physiol. 259:C358-C364 (1990).
Welsh et al., "Abnormal Regulation Of Ion Channels In Cystic Fibrosis Epithelia", FASEB J. 4:2718-2725 (1990).
Welsh et al., "Chloride And Potassium Channels In Cystic Fibrosis Airway Epithelia", Nature 322:467-470 (1986).
White et al., "A Frame-Shift Mutation In The Cystic Fibrosis Gene", Nature 344:655-667 (1990).
Willumsen et al., "Activation Of An Apical Cl.sup.- Conductance By Ca.sup.2+ Ionophores In Cystic Fibrosis Airway Epithelia", Am. J. Physiol. 256:C226-C233 (1989).
Wilson et al., "Correction Of CD18-Deficient Lymphocytes By Retrovirus-Mediated Gene Transfer", Science 248:1413-1416 (1990).
Wilson et al., "Correction Of The Genetic Defect In Hepatocytes From The Watanabe Heritable Hyperlipidemic Rabbit", PNAS (USA)85:4421-4425 (1988).
Wilson et al., "Expression Of Human Adenosine Deaminase In Mice Reconstituted With Retrovirus-Transduced Hematopoietic Stem Cells", PNAS (USA)87:439-443 (1990).
"The Cystic Fibrosis Genetic Analysis Consortium", Am. J. Hum. Genet. 47:354-359 (1990).
Schoumacher et al., "Phosphorylation Fails To Activate Chloride Channels From Cystic Fibrosis Airway Cells", Nature 330:752-754 (1987).
Jetten et al., "Persistence Of Abnormal Chloride Conductance Regulation in Transformed Cystic Fibrosis Epithelia", Science 244:1472 (1989).
Engelhardt, J. F. et al., "Ablation of E2A in Recombinant Adenoviruses Improves Transgene Persistence and Decreases Inflammatory Response in Mouse Liver," PNAS(USA) 91:6196-6200 (1994).
Engelhardt, J. F. et al., "Prolonged Transgene Expression in Cotton Rat Lung with Recombinant Adenoviruses Defective in E2a," Human Gene Therapy 5:1217-1229 (1994).
Goldman, M. J. et al., "Transfer of the CFTR Gene to the Lung of Nonhuman Primates with E1--Deleted, E2a--Defective Recombinant Adenoviruses: a Preclinical Toxicology Study," Human Gene Therapy 6:839-851 (1995).
Goldman, M. J. et al., "Gene Therapy in a Xenograft Model of Cystic Fibrosis Lung Corrects Chloride Transport More Effectively than the Sodium Defect," Nature Genet. 9:126-131 (1995).
Berkner, K. L., "Development of Adenovirus Vectors for the Expression of Heterologous Genes," Biotechniques 6:616-629 (1988).
Engelhardt, J. E. et al., "In Vivo Retroviral Gene Transfer into Human Bronchial Epithelia of Xenografts," J. Clin. Invest. 90:2598-2607 (1992).
Engelhardt, J. F. et al., "Direct gene transfer CFTR into human bronchial epithelia of xenografts with E1-deleted adenoviruses," Nature Genet. 4:27-34 (1993).
Engelhardt, J. F. et al., "Reconstitution of tracheal grafts with a genetically modified epithelium," Proc. Natl. Acad. Sci. (USA) 88:11192-11196 (1991).
Ensinge

Affiliated with

Also associated with

Rating

Adenovirus vectors for gene therapy does not yet have a rating. At this time, there are no reviews or comments for this patent.

If you have personal experience with Adenovirus vectors for gene therapy, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Adenovirus vectors for gene therapy will most certainly appreciate the feedback.

Rate now

Comments { 0 }

Profile ID: LFUS-PAI-O-1991419