Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Radical -xh acid – or anhydride – acid halide or salt thereof...
Patent
1993-06-07
1997-10-07
O'Sullivan, Peter
Drug, bio-affecting and body treating compositions
Designated organic active ingredient containing
Radical -xh acid, or anhydride, acid halide or salt thereof...
514546, 514549, 514552, 514548, 514560, 514826, 514851, 560205, 560265, 562598, 562606, C07C 53126, C07C 53125, C07C 53122, A61K 3119
Patent
active
056748981
ABSTRACT:
Methods and compositions for treating Cystic Fibrosis by mobilizing mutant forms of CFTR, which retain at least some functional activity, to the plasma membrane where they can mediate chloride ion transport are disclosed.
REFERENCES:
patent: 4873316 (1989-10-01), Meade et al.
patent: 5240846 (1993-08-01), Collins et al.
Annunziato et al., "Treatment with Sodium Butyrate." Chromasoma (Berl) 1988 96: 132-138.
McKnight et al. "Butyrate and Related Inhibition of Histone, etc." Cell, vol. 22, 469-477, 1980.
Cheng et al., "Functional Activation, etc." Am. J. Physiology 12(4) 1995 pp. L615-L624.
Boggaram et al., "Regulation of Expression, etc." J. Biol. Chem., vol. 264, No. 19, pp. 11421-11427 (1989).
Bates et al. "Modulation of P-Glycoprotein Phosphorylation, etc." Biochemistry 1992, 31, 6366-6372.
Perrine et al. "Sodium Butyrate Enhances Fetal Globin Gene Expression, etc." Blood, vol. 74, No. 1 (Jul.), 1989: pp. 454-459.
Perrine et al., "Butyric Acid Analogues, etc." Biochemical and Biophysical Research Communication, vol. 148, No. 2, pp. 694-700 (1987).
Novogrodsky et al. "Effect of Polar Organic Compounds on Leukemic Cells" Cancer 51:9-14 (1983).
Nichols et al. "Regulation of Surfactant Protein A, etc" Am. J. Phys. 259, l448-l495, (1990).
Toscani et al. "Sodium Butyrate in Combination with Insulin, etc." The Journal of Biological Chemistry vol. 265, No. 10, pp. 5722-5730, (1990).
Saito et al., "Flow Cytometric and Biochemical Analysis, etc." Cytometry, 12: 757-764 (1991).
Prasad et al. "Mini Review: Butyric Acid, etc." Life Sciences, vol. 27, pp. 1351-1358 (1980).
Peterec, et al. "Butyrate Modulates Surfactant Protein, etc." Am. J. Physiol. 267 (Lung. Cell. Mol. Physiol. 11):L9-L15, (1994).
Annunziato, A.T. et al., "Treatment with Sodium Butyrate Inhibits the Complete Condensation of Interphase Chromatin", Chromosoma, 96:132-138 (1988).
Bates, S.E. et al., "Modulation of P-Glycoprotein Phosphorylation and Drug Transport by Sodium Butyrate", Biochemistry, 31:6366-6372 (1992).
Boggaram, V. et al., "Regulation of Expression of the Gene Encoding the Major Surfactant Protein (SP-A) in Human Fetal Lung in Vitro", The Journal of Biological Chemistry, 264:11421-11427 (1989).
Cheng, S.A. et al., "Functional Activation of the Cystic Fibrosis Trafficking Mutant .increment.F508-CFTR By Overexpression", American Journal of Physiology, 12: L615-L624 (1995).
McKnight, G.S. et al., "Butyrate and Related Inhibitors of Histone Deacetylation Block the Induction of Egg White Genes by Steroid Hormones", Cell, 22: 469-477 (1980).
Nichols, K.V. et al., "Regulation of Surfactant Protein A mRNA By Hormones and Butyrate in Cultured Fetal Rat Lung", American Journal of Physiology, 259:L448-L495 (1990).
Nichols, K.V. et al., "Inhibition of SP-A mRNA By Butyric Acid Analogs In Fetal Rat Lung Explants", Pediatric Research, 27: 49A (1990).
Novogrodsky, A. et al., "Effect of Polar Organic Compounds on Leukemic Cells", Cancer, 51: 9-14 (1983).
Perrine, S.P. et al., "Butyric Acid Analogues Augment y Globin Gene Expression in Neonatal Erythroid Progenitors", Biochemical and Biophysical Research Communications, 148: 694-700 (1987).
Perrine, S.P. et al., "Sodium Butyrate Enhances Fetal Globin Gene Expression in Erythroid Progenitors of Patients With HbSS and .beta. Thalassemia", Blood, 74:454-459 (1989).
Peterec, S.M. et al., "Butyrate ModulatesSurfactant Protein mRNA in Fetal Rat Lung By Altering mRNA Transcription and Stability", American Journal of Physiology, 11: L9-L15 (1994).
Prasad, K.N., "Butyric Acid: A Small Fatty Acid With Diverse Biological Functions", Life Sciences, 27: 1351-1358 (1980).
Saito, S.et al., "Flow Cytometric and Biochemical Analysis of Dose-Dependent Effects of Sodium Butyrate on Human Endometrial Adenocarcinoma Cells", Cytometry, 12:757-764 (1991).
Toscani, A. et al., "Sodium Butyrate in Combination With Insulin or Dexamethasone Can Terminally Differentiate Actively Proliferating Swiss 3T3 Cells Into Adipocytes" The Journal of Biological Chemistry, 265: 5722-5730 (1990).
Drumm, M.L. et al. (1991) "Chloride Conductance Expressed by .increment.F508 and Other Mutant CFTRs in Xenopus Oocytes" Science 254: 1797-1799.
Machamer, C.E. (1988) "Vesicular Stomatitis Virus G Proteins With Altered Glycosylation Sites Display Temperature-Sensitive Intracellular Transport and are Subject To Abberant Intermolecular Disulfide Bonding" J. Biol. Chem. 263:5955-5960;.
J. Marshall et al., "Stoichiometry of Recombinant Cystic Fibrosis Transmembrane Conductance Regulator in Epithelial Cells and Its Functional Reconstitution into Cells in vitro", The Journal of Biological Chemistry, 269, 1994, pp. 2987-2995.
J. Amara et al., "Intracellular protein trafficking defects in human disease", Trends in Cell Biology, 2, 1992, pp. 145-149.
C. Li et al., "The cystic fibrosis mutation (.increment.F508) does not influence the chloride channel activity of CFTR", Nature Genetics, 3, 1993, pp. 311-316.
W. Dalemans et al., "Altered chloride ion channel kinetics associated with the .increment.F508 cystic fibrosis mutation", Nature, 354, 1991, pp. 526-528.
M. Drumm et al., "Chloride Conductance Expressed by .increment.F508 and Other Mutant CFTRs in xenopus Oocytes", Science, 254, 1991, pp. 1797-1799.
C.Machamer et al., "Vesicular Stomatitis Virus G Proteins with Altered Glycosylation Sites Display Temperature-sensitive Intracellular Transport and Are Subject to Aberrant Intermolecular Disulfide Bonding", The Journal of Biological Chemistry, 263, 1988, pp. 5955-5960.
Andrews, G.K. et al. (1987) "Butyrate Selectively Activates the Metallothionein Gene in Teratocarcinoma Cells and Induces Hypersensitivity to Metal Induction" Nucleic Acids Res. 15(13):5461-5475.
Rommens, J.H. et al. (1989) "Identification of the Cystic Fibrosis Gene: Chromosome Walking and Jumping" Science 245: 1059-1065.
Kerem, B.S. et al. (1989) "Identification of the Cystic Fibrosis Gene: Genetic Analysis" Science 245:1073-1080.
Dorin, J.R. et al. (1987) "A Clue to the Basic Defect in Cystic Fibrosis from Cloning the CF Antigen Gene" Nature 326:614-617;.
Kerem, B-S. et al. (1990) "Identification of Mutations in Regions Corresponding to the Two Putative Nucleotide (ATP)-Binding Folds of the Cystic Fibrosis Gene" Proc. Natl. Acad. Sci. 87:8447-8451;.
Cheng. S. H. et al. (1990) "Defective Intracellular Transport and Processing of CFTR is the Molecular Basis of Most Cystic Fibrosis" Cell 63:827-834;.
Riordan, J.R. et al. (1989) "Identification of the Cystic Fibrosis Gene: Cloning and Characterization of Complementary DNA" Science 245:1066-1073;.
Drumm, M.L. et al. (1990) "Correction of the Cystic Fibrosis Defect In Vitro By Retrovirus-Mediated Gene Transfer" Cell 62:1227-1233;.
Gregory, R.J. et al. (1990) "Expression and Characterization of the Cystic Fibrosis Transmembrane Conductance Regulator" Nature 347:382-386;.
Rich, D.P. et al. (1990) "Expression of the Cystic Fibrosis Transmembrane Conductance Regulator Corrects Defective Chloride Channel Regulation in Cystic Fibrosis Airway Epithelial Cells" Nature 347:358-363;.
Dalemans, W. et al. (1991) "Altered Chloride Ion Channel Kinetics Associated with the .increment.F508 Cystic Fibrosis Mutation" Nature 354:526-528;.
Dean, M. et al. (1990) "Multiple Mutations in Highly Conserved Residues Are Found in Mildly Affected Cystic Fibrosis Patients" Cell 61:863-870;.
Cutting, G.R. et al. (1990) "A Cluster of Cystic Fibrosis Mutations in the First Nucleotide-Binding Fold of the Cystic Fibrosis Conductance Regulator Protein" Nature 346:366-369;.
Cheng, S. H. et al. (1991) "Phosphorylation of the R Domain by cAMP-Dependent Protein Kinase Regulates the CFTR Chloride Channel" Cell 66:1027-1036;.
Anderson, M.P. et al. (1991) "Nucleoside Triphosphates are Required to Open the CFTR Chloride Channel" Cell 67:775-784;.
Hamosh, A. et al. (1991) "Severe Deficiency of Cystic Fibrosis Transmembrane Conductance Regulator Messenger RNA Carrying Nonsense Mutations R553X and W1316X in Respiratory Epithelial Cells of Patients with Cystic Fibrosis" J. Clin. Invest. 88: 1880-1885;.
Rich, D.P. et al. (1991) "Effect of Deleting the R Domain on CFTR
Cheng Seng Hing
Fang Shaona Lee
Hoppe, IV Henry
Smith Alan Edward
Genzyme Corporation
O'Sullivan Peter
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