Recombinant proteins for genetic disease

Clinical Genetics

Volumn 55, Issue 6, 1999, Pages 389-394

  Russell, Chris S ^a,b   Clarke, Lorne A ^a  

^a UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^b NONE   (Canada)

Author keywords

Fusion proteins; Genetic disease; Protein targeting; Recombinant proteins

Indexed keywords

ADENOSINE DEAMINASE; ALGLUCERASE; ALPHA GALACTOSIDASE; ALPHA GLUCOSIDASE; ALTEPLASE; BETA GLUCURONIDASE; BLOOD CLOTTING FACTOR 9; CYTOKINE; DEOXYRIBONUCLEASE; GLUCOSYLCERAMIDASE; HUMAN GROWTH HORMONE; HYBRID PROTEIN; IMIGLUCERASE; INTERFERON BETA SERINE; LEPTIN; LEVO IDURONIDASE; N ACETYLGALACTOSAMINE 6 SULFATASE; N4 (BETA N ACETYLGLUCOSAMINYL)ASPARAGINASE; PHENYLALANINE AMMONIA LYASE; PROTEIN P53; RECOMBINANT BLOOD CLOTTING FACTOR 8; RECOMBINANT ENZYME; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; RECOMBINANT HUMAN INSULIN; RECOMBINANT NERVE GROWTH FACTOR; RECOMBINANT PROTEIN; SUPEROXIDE DISMUTASE; TRYPSIN INHIBITOR; TUMOR NECROSIS FACTOR ANTIBODY;

BLOOD BRAIN BARRIER; COMBINED IMMUNODEFICIENCY; DIABETES MELLITUS; DRUG COST; DRUG DELIVERY SYSTEM; DRUG MANUFACTURE; DRUG MECHANISM; DRUG PURIFICATION; ENZYME REPLACEMENT; GAUCHER DISEASE; GENETIC DISORDER; HUMAN; HURLER SYNDROME; IMMUNOPATHOLOGY; INTRACEREBRAL DRUG ADMINISTRATION; INTRACEREBROVENTRICULAR DRUG ADMINISTRATION; NONHUMAN; PHENYLKETONURIA; PRIORITY JOURNAL; PROTEIN TARGETING; PROTEIN TRANSPORT; REVIEW;

ANIMALS; GENETIC DISEASES, INBORN; HUMANS; RECOMBINANT PROTEINS;

EID: 0032793110     PISSN: 00099163     EISSN: None     Source Type: Journal    
DOI: 10.1034/j.1399-0004.1999.550601.x     Document Type: Review

References (34)

1. Safos S, Chang TM. Enzyme replacement therapy in ENU2 phenylketonuric mice using oral microencapsulated phenylalanine ammonia-lyase: a preliminary report. Artif Cells Blood Substit Immobil Biotechnol 1995: 23: 681-692.
2. Sarkissian CN, Shao Z, Blain F, Peevers R, Su H, Heft R, Chang TM, Scriver CR. A different approach to treatment of phenylketonuria: phenylalanine degradation with recombinant phenylalanine ammonia lyase. Proc Natl Acad Sci USA 1999: 6: 2339-2344.
3. Bates RD, Nahata MC. Aerosolized dornase alpha (rhDNase) in cystic fibrosis. J Clin Pharm Ther 1995: 20: 313-315.
4. Pfeffer SR. Targeting of proteins to the lysosome. Curr Top Microbiol Immunol 1991: 170: 43-63.
5. Niederau C, vom Dahl S, Haussinger D. First long-term results of imiglucerase therapy of type 1 Gaucher disease. Eur J Med Res 1998: 3: 25-30.
6. Grabowski GA, Barton NW, Pastores G, Dambrosia JM, Banerjee TK, McKee MA, Parker C, Schiffmann R, Hill SC, Brady RO. Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources. Ann Intern Med 1995: 122: 33-39.
7. http://www.genzyme.com/prodserv/specialty_therapeutics/ cerezyme/welcome.htm
8. Brady RO, Murray GJ, Barton NW. Modifying exogenous glucocerebrosidase for effective replacement therapy in Gaucher disease. J Inherit Metab Dis 1994: 17: 510-519.
9. Hermann G, Pastores GM, Abdelwahab IF. Lorberboym AM. Gaucher disease: assessment of skeletal involvement and therapeutic responses to enzyme replacement. Skeletal Radiol 1997: 26: 687-696.
10. Sells MA, Li J, Chernoff J. Delivery of protein into cells using polycationic liposomes. Biotechniques 1995: 19: 72-76.
11. Park E, Starzyk RM, McGrath JP, Lee T, George J, Schutz AJ, Lynch P, Putney SD. Production and characterization of fusion proteins containing transferrin and nerve growth factor. J Drug Target 1998: 6: 53-64.
12. Ashkenazi A, Chamow SM. Immunoadhesins as research tools and therapeutic agents. Curr Opin Immunol 1997: 9: 195-200.
13. Moreland LW, Baumgartner SW, Schiff MH, Tindall EA, Fleischmann RM, Weaver AL, Ettlinger RE, Cohen S, Koopman WJ, Mohler K, Widmer MB, Blosch CM. Treatment of rheumatoid arthritis with a recombinant human tumor necrosis factor receptor (p75)-Fc fusion protein. N Engl J Med 1997: 337: 141-147.
14. Saleh MN, LeMaistre CF, Kuzel TM, Foss F, Platanias LC, Schwartz G, Ratain M, Rook A, Freytes CO, Craig F, Reuben J, Sams MW, Nichols JC. Antitumor activity of DAB389IL-2 fusion toxin in mycosis fungoides. J Am Ac Derm 1998: 39: 63-73.
15. Elliott G, O'Hare P. Intercellular trafficking and protein delivery by a herpes virus structural protein. Cell 1997: 88: 223-233.
16. Elliott G, O'Hare P. Intercellular trafficking of VP22-GFP fusion proteins. Gene Therapy 1999: 6: 149-151.
17. Phelan A, Elliott G, O'Hare P. Intercellular delivery of functional p53 by the herpes virus protein VP22. Nat Biotech 1998: 16: 440-443.
18. Figueiredo DM, Hallewell RA, Chen LL, Fairweather NF, Dougan G, Savitt JM, Parks DA, Fishman PS. Delivery of recombinant tetanus-superoxide dismutase proteins to central nervous system neurons by retrograde axonal transport. Exper Neur 1997: 145: 546-554.
19. Kroll RA, Neuwelt EA. Outwitting the blood-brain barrier for therapeutic purposes: osmotic opening and other means. Neurosurgery 1998: 42: 1099-1100.
20. Smith RA, Balis FM, Ott KH, Elsberry DD, Sherman MR, Saifer MG. Parmacokinetics and tolerability of ventricularly administered superoxide dismutase in monkeys and preliminary clinical observations in familial ALS. J Neurol Sci 1995: 129: 13-18.
21. Zirzow GC, Sanchez OA, Murray GJ, Brady RO, Oldfield EH. Delivery, distribution, and neuronal uptake of exogenous mannose-terminal glucocerebrosidase in the intact rat brain. Neurochem Res 1999: 24: 301-305.
22. Kakkis ED, McEntee MF, Schmidtchen A, Neufeld EF, Ward DA, Gompf RE, Kania S, Bedolla C, Chien SL, Shull RM. Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I. Biochem Mol Med 1996: 58: 156-167.
23. Vogler C, Sands MS, Levy B, Galvin N, Birkenmeier EH, Sly WS. Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival. Pediatr Res 1996: 39: 1050-1054.
24. Torzilli PA, Arduino JM, Gregory JD, Bansal M. Effect of proteoglycan removal on solute mobility in articular cartilage. J Biomech 1997: 30: 895-902.
25. Brooks DA, Hopwood JJ, King BM. Immune response to enzyme replacement therapy: clinical signs of hypersensitivity reactions and altered enzyme distribution in a high titre rat model. Biochim Biophys Acta 1998: 14: 163-172.
26. Vial T, Descotes J. Immune-mediated side-effects of cytokines in humans. Toxicology 1995: 105: 31-57.
27. Grabowski GA, Leslie N, Wenstrup R. Enzyme therapy for Gaucher disease: the first 5 years. Blood Rev 1998: 12: 115-133.
28. Colman A. Production of therapeutic proteins in the milk of transgenic livestock. Biochem Soc Symp 1998: 63: 141-147.
29. Haq TA, Mason HS, Clements JD, Arntzen CJ. Oral immunization with a recombinant bacterial antigen produced in transgenic plants. Science 1995: 268: 714-716.
30. Tacket CO, Mason HS, Losonsky G, Clements JD, Levine MM, Arntzen CJ. Immunogenicity in humans of a recombinant bacterial antigen delivered in a transgenic potato. Nat Med 1998: 4: 607-609.
31. van Rooijen GJ, Moloney MM. Plant seed oil-bodies as carriers for foreign proteins. Biotechnology 1995: 13: 72-77.
32. Schmidt M, Babu KR, Khanna N, Marten S, Rinas U. Temperature-induced production of recombinant human insulin in high-cell density cultures of recombinant Escherichia coli. J Biotechnol 1999: 68: 71-83.
33. Fawzi AB, Zhang H, van Heek M, Graziano MP. Purification of milligram quantities of human leptin from recombinant E. coli. Horm Metab Res 1996: 28: 694-697.
34. Belew M, Zhou Y, Wang S, Nystrom LE. Purification of recombinant human granulocyte-macrophage colony-stimulating factor from the inclusion bodies produced by transformed Escherichia coli cells. J Chromat A 1994: 679: 67-83.