Chemistry: natural resins or derivatives; peptides or proteins; – Proteins – i.e. – more than 100 amino acid residues – Blood proteins or globulins – e.g. – proteoglycans – platelet...
Patent
1997-09-23
1999-01-19
Carlson, Karen Cochrane
Chemistry: natural resins or derivatives; peptides or proteins;
Proteins, i.e., more than 100 amino acid residues
Blood proteins or globulins, e.g., proteoglycans, platelet...
435 691, C07K 14805, C12P 2106
Patent
active
058614885
ABSTRACT:
Gene therapy methods and compositions for high level expression of anti-sickling globin proteins in erythroid cells for treating Sickle Cell disease are described.
REFERENCES:
patent: 5126260 (1992-06-01), Tuan et al.
Stamatoyannopoulos, George, M.D., Dr. Sci, et al., "Regulatory Elements of the Individual Globin Genes", The Molecular Basis of Blood Diseases (Second Edition), W.B. Saunders Company, pp. 116-120.
LeBoulch, Philippe, et al., "Mutagenesis of Retroviral Vectors Transducing Human .beta.-Globin Gene and .beta.-Globin Locus Control Region Derivatives Results in Stable Transmission of an Active Transcriptional Structure", The EMBO Journal, vol. 13, No. 13, (1994) pp. 3065-3076.
Kosche, Katherine A., "DNA Sequences Regulating Human .beta. Globin Gene Expression", Nucleic Acids Research, vol. 13, No. 21 (1985), pp. 7781-7793.
Nagel, Ronald L., "Structural Bases of the Inhibitory Effects of Hemoglobin F and Hemoglobin A.sub.2 on the Polymerization of Hemoglobin S", Proc. Natl. Acad. Sci, USA, vol. 76, No. 2 (Feb. 1979), pp. 670-672.
Lawn et al. 1980 Cell 21: 647-651.
Spritz et al 1980 Cell 21: 639-646.
Goodman et al 1983 Nature 303:546-548.
Nagai et al 1995 Blood 86(10):998A.
Curd, John G., et al., "Antibodies to an NH.sub.2 -terminal Fragment of .beta..sup.S Globin", The Journal of Biological Chemistry, vol. 251, No. 5 (Mar. 10, 1976) pp. 1290-1295.
Eastlake, Ann, et al., "The NH.sub.2 -terminal Region of the .beta. Chain of Sickle Hemoglobin", The Journal of Biological Chemistry, vol. 251, No. 20 (Oct. 25, 1976) pp. 6426-6430.
Enver, Tariq, et al., "The Human .beta.-globin Locus Activation Region Alters the Development Fate of a Human Fetal Globin Gene in Transgenic Mice", Proc. Natl. Acad. Sci, USA, vol. 86, (Sep. 1989) pp. 7033-7037.
McCune Steven L., et al., "Recombinant Human Hemoglobins Designed for Gene Therapy of Sickle Cell Disease" Proc. Natl. Acad. Sci., USA, vol. 91, (Oct. 1994), pp. 9852-9856.
Otieno, Samuel, et al., "Specific Antibodies to Hemoglobin A.sub.1 (ant-Glu) and Hemoglobin S (anti-Val) in the Guinea Pig: Immunologic and Structural Correlations.sup.1 ", The Journal of Immunology, vol. 121, No. 6 (Dec. 1978), pp. 2458-2462.
Roth, Eugene F. Jr., et al., "Chemical Modification of Human Hemoglobin by Antisickling Concentrations of Nitrogen Mustard", Blood, vol. 58, No. 2 (Aug. 1981), pp. 300-308.
Takekoshi, Ken J., et al., "Retroviral Transfer of a Human .beta.-globin/.delta.globin Hybrid Gene Linked to .beta. Locus Control Region Hypersensitive Site 2 Aimed at the Gene Therapy of Sickle Cell Disease", Proc. Natl. Acad. Sci., USA, vol. 92, No. 7 (Mar. 1995) pp. 3014-3018.
Leboulch Philippe
London Irving M.
Carlson Karen Cochrane
Engellenner Thomas J.
Massachusetts Institute of Technology
Remillard Jane E.
LandOfFree
Compositions and methods for treating sickle cell disease does not yet have a rating. At this time, there are no reviews or comments for this patent.
If you have personal experience with Compositions and methods for treating sickle cell disease, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Compositions and methods for treating sickle cell disease will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-1247568