Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Carbohydrate doai
Patent
1995-10-11
1998-05-05
Stone, Jacqueline M.
Drug, bio-affecting and body treating compositions
Designated organic active ingredient containing
Carbohydrate doai
514182, 514777, 552544, 560 6, A61K 4800, A61K 3156, A61K 3170, B01F 1728
Patent
active
057474710
ABSTRACT:
Novel cationic amphiphiles are provided that facilitate transport of biologically active (therapeutic) molecules into cells. The amphiphiles contain lipophilic groups derived from steroids, from mono or dialkylamines, or from ether or ester-linked alkyl groups, and cationic groups, protonatable at physiological pH, derived from amines, alkylamines or polyalkylamines. There are provided also therapeutic compositions prepared typically by contacting a dispersion of one or more cationic amphiphiles with the therapeutic molecules. Therapeutic molecules that can be delivered into cells according to the practice of the invention include DNA, RNA, and polypeptides. Representative uses of the therapeutic compositions of the invention include providing gene therapy, and delivery of antisense polynucleotides or biologically active polypeptides to cells. With respect to therapeutic compositions for gene therapy, the DNA is provided typically in the form of a plasmid for complexing with the cationic amphiphile. Novel and highly effective plasmid constructs are also disclosed, including those that are particularly effective at providing gene therapy for clinical conditions complicated by inflammation.
REFERENCES:
patent: 4394448 (1983-07-01), Szoka et al.
patent: 4544545 (1985-10-01), Ryan et al.
patent: 4897355 (1990-01-01), Eppstein et al.
patent: 4958013 (1990-09-01), Letsinger
patent: 4971803 (1990-11-01), Li et al.
patent: 5004737 (1991-04-01), Kim et al.
patent: 5171678 (1992-12-01), Behr et al.
patent: 5264618 (1993-11-01), Felgner et al.
patent: 5283185 (1994-02-01), Epand et al.
patent: 5334761 (1994-08-01), Gebeyehu et al.
patent: 5416203 (1995-05-01), Letsinger
Leventis et al., "Interactions of Mammalian Cells With Lipid Dispersions Containing Novel Metabolizable Cationic Amphiphiles", Biochimica et Biophysica Acta, 1023, 1990, pp. 124-132.
K. Patel et al., "Modification of Vesicle Surfaces with Amphiphilic Sterols. Effect on Permeability and In Vivo Tissue Distribution", Biochimica et Biophysica Acta, 814, 1985, pp. 256-264.
K. Moore et al., "Squalamine: An Aminosterol Antibotic from the Shark", Proceedings of the National Academy of Sciences USA, 90, 1993, pp. 1354-1358.
J. Guy-Caffey et al., "Novel Polyaminolipids Enhance the Cellular Uptake of Oligonucleotides", The Journal of Biological Chemistry, 270, 1995, pp. 31391-31396.
J. Janne, et al., "Polyamines: From Molecular Biology to Clinical Applictions", Annals of Medicine, 23, 1991, pp. 241-259.
N. Caplen, et al., "Liposome Mediated CFTR Gene Transfer to the Nasal Epithelium of Patients with Cystic Fibrosis", Nature Medicine, 1, 1995, pp. 39-46.
J-S Remy et al., "Gene Transfer with a Series of Lipophilic DNA Binding Molecules", Bioconjugate Chemistry, 5, 1994, pp. 647-654.
J-P. Behr, "Gene Transfer with Synthetic Cationic Amphiphiles: Prospects for Gene Therapy", Bioconjugate Chemistry, 5, 1994, pp. 382-389.
J. Felgner, et al., "Enhanced Gene Delivery and Mechanism Studies with a Novel Series of Cationic Lipid Formulations", The Journal of Biological Chemistry, 269, 1994, pp. 2550-2561.
F. Barthel et al., "Gene Transfer Optimzation with Lipospermine-Coated DNA", DNA and Cell Biology, 12, 1993, pp. 553-560.
J-P. Behr, "Synthetic Gene-Transfer Vectors", Accounts of Chemical Research, 26, 1993, pp. 274-278.
P. Hoet, et al., "Kinetics and Cellular Localization of Putrescine Uptake in Human Lung Tissue", Thorax, 48, 1993, pp. 1235-1241.
J. Felgner, et al., "Cationic Lipid-Mediated Delivery of Polynucleotides", Methods (A Companion to Methods in Enzymology), 5, 1993, pp. 67-75.
X. Gao, et al., "A Novel Cationic Liposome Reagent for Efficient Transfection of Mammalian Cells", Biochemical and Biophysical Research Communications, 179, 1991, pp. 280-285.
J. Rose, et al., "A New Cationic Liposome Reagent Mediating Nearly Quantitative Transfection of Animal Cells", Biotechniques, 10, 1991, pp. 520-525.
J. Loeffler, et al., "Lipopolyamine-Mediated Transfection Allows Gene Expression Studies in Primary Neuronal Cells", Journal of Neurochemistry, 54, 1990, pp. 1812-1815.
J. Cheetham, et al., "Cholesterol Sulfate Inhibits the Fusion of Sendai Virus to Biological and Model Membranes", The Journal of Biological Chemistry, 265, 1990, pp. 12404-12409.
J-P. Behr, et al., "Efficient Gene Transfer into Mammalian Primary Endocrine Cells with Lipopolyamine-Coated DNA", Proceedings of the National Academy of Sciences, USA, 86, 1989, pp. 6982-6986.
R. Letsinger, et al., "Cholesteryl-Conjugated Oligonucleotides: Synthesis, Properties, and Activity as Inhibitors of Replication of Human Immunodeficiency Virus in Cell Culture", Proceedings of the National Academy of Sciences, USA, 86, 1989, pp. 6553-6556.
L. Stamatos, et al., "Interactions of Cationic Lipid Vesicles with Negatively Charged Phospholipid Vesicles and Biological Membranes", Biochemistry, 27, 1988, pp. 3917-3925.
P. Felgner, et al., "Lipofection: A Highly Efficient, Lipid-Mediated DNA-Transfection Procedure", Proceedings of the National Acadmeny of Sciences, USA, 84, 1987, pp. 7413-7417.
R. Rando, et al., "The Synthesis and Properties of a Functional Fluorescent Cholesterol Analog", Biochemica et Biophysica Acta, 684, 1982, pp. 12-20.
A. Pegg, "Polyamine Metabolism and its Importance in Neoplastic Growth and as a Target for Chemotherapy", Cancer Research, 48, 1988, pp. 759-774.
R. Kameji, et al., "Spermidine Uptake by Type II Pulmonary Epithelial Cells in Primary Cells in Primary Culture", American Journal of Physiology, 256, 1989, pp. C161-C167.
Cheng Seng H.
Eastman Simon J.
Harris David J.
Hubbard Shirley C.
Lane Mathieu B.
Donahue E. Victor
Genzyme Corporation
Stone Jacqueline M.
Twomey Patrick
LandOfFree
Cationic amphiphiles containing steroid lipophilic groups for in does not yet have a rating. At this time, there are no reviews or comments for this patent.
If you have personal experience with Cationic amphiphiles containing steroid lipophilic groups for in, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Cationic amphiphiles containing steroid lipophilic groups for in will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-54702