Chemistry: molecular biology and microbiology – Treatment of micro-organisms or enzymes with electrical or... – Modification of viruses
Patent
1994-05-19
1998-10-27
Rories, Charles C.P.
Chemistry: molecular biology and microbiology
Treatment of micro-organisms or enzymes with electrical or...
Modification of viruses
435 6, 435 691, 435325, 435354, 435375, 424417, 424420, 424450, 514 44, 800 2, C12N 1564, A61K 9127, A61K 4800, C12Q 168
Patent
active
058277030
ABSTRACT:
Novel methods and compositions are provided for introducing a gene capable of modulating the genotype and phenotype into two or more tissues following systemic administration. The gene can be introduced into a mammalian host by way of an expression vector either as naked DNA or complexed to lipid carriers, particularly cationic lipid carriers. Multiple individual tissues can be transfected using naked DNA. Using a DNA: lipid carrier complex, multiple tissues and cell types can be transfected. The techniques and compositions find use in the palliation or treatment of any of a variety of genetic-based disorders.
REFERENCES:
patent: 3826255 (1974-07-01), Havstad et al.
patent: 4046146 (1977-09-01), Rosskamp et al.
patent: 4253468 (1981-03-01), Lehmbeck
patent: 4268460 (1981-05-01), Boiarski et al.
patent: 4394448 (1983-07-01), Szoka
patent: 4510929 (1985-04-01), Bordoni et al.
patent: 4649911 (1987-03-01), Knight et al.
patent: 4804678 (1989-02-01), Augstein et al.
patent: 4946787 (1990-08-01), Eppstein et al.
patent: 5032407 (1991-07-01), Wagner
patent: 5075229 (1991-12-01), Hanson
patent: 5240842 (1993-08-01), Mets
patent: 5240846 (1993-08-01), Collins
patent: 5264618 (1993-11-01), Felgner et al.
Dzau, Victor J., et al. (1993) "Gene therapy for cardiovascular disease", TIBTECH 11:205-210.
Friedmann, Theodore (1989) "Progress Toward Human Gene Therapy", Science 244:1275-1281.
Zhu, Ning, et al., (1993) "Systemic Gene Expression After Intravenous DNA Delivery into Adult Mice", Science, 261:209-211.
Rosenfeld, et al. (1992) "In vivo Transfer of the Human Cystic Fibrosis Transmembrane Conductance Regulator Gene to the Airway Epithelium", Cell, 68:143-155.
Brigham, et al. (1989) "Expression of a Prokaryotic Gene in Cultured Lung Endothelial Cells after Lipofection with a Plasmid Vector", Am. J. Respir. Cell. Mol. Biol., 1:95-100.
Alton, E., et al. (1993) "Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice", Nature Genetics, 5:135-142.
Huang, et al. (1990) "The Simian Virus 40 Small-t Intron, Present in Many Common Expression Vectors, Leads to Aberrant Splicing", Molecular and Cellular Biology, 10(4):1805-1810.
Papahadjopoulos et al., "Cochleate Lipid Cylinders: Formation by Fusion of Unilamellar Lipid Vesicles," Biochimica et Biophysica Acta, vol. 394:483-491 (1975).
Deamer et al., "Large Volume Liposomes by an Ether Vaporization Method," Biochimica et Biophysica Acta, vol. 443:629-634 (1976).
Ostro, et al., "Incorporation of High Molecular Weight RNA into Large Artificial Lipid Vesicles," Biochemical and Biophysical Research Communications, vol. 76, No. 3:836-842 (1977).
Enoch, et al., "Formation and Properties of 1000-A-Diameter, Single-Bilayer Phospholipid Vesicles," PNAS (USA), vol. 76, No. 1:145-149 (1979).
Wilson, et al., "The Introduction of Poliovirus RNA into Cells via Lipid Vesicles (Liposomes), " Cell, vol. 17:77-84 (1979).
Fraley, et al., "Entrapment of a Bacterial Plasmid in Phospholipid Vesicles: Potential for Gene Transfer," PNAS (USA), vol. 76, No. 7:3348-3352 (1979).
Huang, et al., "Monoclonal Antibody Covalently Coupled with Fatty Acid," Journal of Biological Chemistry, vol. 255, No. 17:8015-8018 (1980).
Fraley, et al., "Introduction of Liposome-Encapsulated SV40 DNA into Cells," Journal of Biological Chemistry, vol. 255 No. 21:10431-10435 (1980).
Leserman, et al., "Targeting to Cells of Flourescent Liposomes Covalently Coupled with Monoclonal Antibody of Protein A," Nature, vol. 288:602-604 (1980).
Beaucage, et al., "Deoxynucleoside Phosphoramidites--A New Class of Key Intermediates for Deoxypolynucleotide Synthesis," Tetrahedron Letters, vol. 22 No. 20:1859-1862 (1981).
Duckworth, et al., "Rapid Synthesis of Oligodeoxyribonucleotides VI. Efficient, Mechanised Synthesis of Heptadecadeoxyribonucleotides by an Improved Solid Phase Phosphotriester Route," Nucleic Acids Research, vol. 9 No. 7:1691-1706 (1981).
Martin, et al., "Immunospecific Targeting of Liposomes to Cells: A Novel and Efficient Method for Covalent Attachment of Fab' Fragments via Disulfide Bonds," Biochemistry, vol. 20:4229-4238 (1981).
Matteucci, et al., "Synthesis of Deoxyoligonucleotides on a Polymer Support," J. Am. Chem. Soc., vol. 103:3185-3191 (1981).
Volloch, et al., "Stability of Globin mRNA in Terminally Differentiating Murine Erythroleukemia Cells," Cell vol. 23:509-514 (1981).
Bothwell, et al., "Heavy Chain Variable Region Contribution to the NP Family of Antibodies: Somatic Mutation Evident in a .gamma.a Variable Region," Cell, vol. 24:625-637 (1981).
Edge, et al., "Total Synthesis of a Human Leukocyte Interferon Gene," Nature, vol. 292:756-762 (1981).
Schaefer-Ridder, et al., "Liposomes as Gene Carriers: Efficient Transformation of Mouse L Cells by Thymidine Kinase Gene," Science, vol. 215:166-168 (1981).
Gorman, et al., "Recombinant Genomes which Express Chloramphenicol Acetyltransferase in Mammalian Cells," Molecular and Cellular Biology, vol. 2 No. 9:1044-1051 (1982).
Gorman, et al., "The Rous Sarcoma Virus Long Terminal Repeat is a Strong Promoter when Introduced into a Variety of Eukaryotic Cells by DNA-Mediated Transfection," PNAS (USA), vol. 79:6777-6781 (1982).
Long, et al., "Complete Sequence of the cDNA for Human .alpha.-Antitrypsin and the Gene for the S. Variant," Biochemistry, vol. 23:4828-4837 (1984).
Nambair, et al., "Total Synthesis and Cloning of a Gene Coding for the Ribonuclease S. Protein," Science, vol. 223:1299-1301 (1984).
Jay, et al., "Chemical Synthesis of a Biologically Active Gene for Human Immune Interferon-.gamma.," Journal of Biological Chemistry, vol. 259, No. 10:6311-6317 (1984).
Kunkel, Thomas, "Rapid and Efficient Site-specific Mutagenesis without Phenotypic Selection," PNAS (USA), vol. 82:488-492 (1985).
Boshart, et al., "A Very Strong Enhancer is Located Upstream of an Immediate Early Gene of Human Cytomegalovirus," Cell, vol. 41:521-530 (1985).
Stinski, et al., "Activation of the Major Immediate Early Gene of Human Cytomegalovirus by cis-Acting Elements in the Promoter-Regulatory Sequence and by Virus-specific Trans-acting Components," Journal of Virology, vol. 55 No. 2:431-441 (1985).
Cullen, B.R., "Trans-Activation of Human Immunodeficiency Virus Occurs via a Bimodal Mechanism," Cell, vol. 46:973-982 (1986).
Benvenistry, et al., "Direct Introduction of Genes into Rats and Expression of the Genes," PNAS (USA), vol. 83:9551-9555 (1986).
Debs, et al., "Successful Treatment with Aerosolized Pentamidine of Pneumocystis Carinii Pneumonia in Rats," Antimicrobial Agents and Chemotherapy, vol. 31 No. 1:37-41 (1987).
Montgomery, et al., "Aerosolised Pentamidine as Sole Therapy for Pneumocystis Carini Pneumonia in Patients with Acquired Immunodeficiency Syndrome," Lancet, vol. 11:480-483 (1987).,
Felgner, et al., "Lipofection: A Highly Efficient, Lipid-Mediated DNA-Transfection Procedure," PNAS (USA), vol. 84:7413-7417 (1987).
Wang, et al., "pH Sensitive Immunoliposomes Mediate Target-Cell-Specific Delivery and Controlled Expression of a Foreign Gene in a Mouse," PNAS (USA), vol. 84:7851-7855 (1987).
Mannino, et al., "Liposome Mediated Gene Transfer," Biotechniques, vol. 6 No. 7:682-690 (1988).
Sakai, et al., "Hormone-Mediated Repression: A Negative Clucocorticoid Response Element from the Bovine Prolactin Gene," Genes and Development, vol. 2:1144-1154 (1988).
Stamatatos, et al., "Interactions for Cationic Lipid Vesicles with Negatively Charged Phospholipid Vesicles and Biological Membranes," Biochemistry, vol. 27:3917-3925 (1988).
Debs, et al., "Lung-Specific Delivery of Cytokines Induces Sustained Pulmonary and Systemic Immunomodulation in Rats," J. Immunology, vol. 140 No. 10:3482-3488 (1988).
Wu, et al., "Receptor-Mediated Gene Delivery and Expression In Vivo," J. Biological Chemistry, vol. 263, No. 29:14621-14624 (1988).
Hubbard, et al., "Fate of Aerosolized Recombinant DNA-Produced .alpha.1-Antitrypsin: Use of the Epithelial Surface of the Lower Respiratory Tract to Administer Proteins of Therapeutic Importance," PNAS (USA) vol. 86:680-684 (1989).
Kaneda, et al., "Increased Expression of DNA
Debs Robert James
Zhu Ning
Rories Charles C.P.
The Regents of the University of California
LandOfFree
Methods and composition for in vivo 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 Methods and composition for in vivo gene therapy, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Methods and composition for in vivo gene therapy will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-1613156