Fluorescence-based Selection of Retrovirally Transduced Cells in Congenital Erythropoietic Porphyria: Direct Selection Based on the Expression of the Therapeutic Gene

Journal of Gene Medicine

Volumn 1, Issue 5, 1999, Pages 322-330

  Fontanellas, Antonio ^a   Mazurier, Frédéric ^a   Belloc, Francis ^b   Taine, Laurence ^a,c   Dumain, Patrice ^b   Morel, Carine ^a   Ged, Cécile ^a   De Verneuil, Hubert ^a,d   Moreau Gaudry, François ^a  

^a INSERM   (France)

^b Hop du Haut Lévêque   (France)

^c HÔPITAL PELLEGRIN   (France)

^d UNIVERSITÉ DE BORDEAUX   (France)

Author keywords

Flow cytometry cell sorting; Fluorescence based selection; Gene therapy; Gene transfer; Porphyria; Retrovirus

Indexed keywords

UNIDENTIFIED RETROVIRUS;

AMINOLEVULINIC ACID; MELATONIN; PORPHYRIN;

ADULT; ARTICLE; CELL LINE; CELL SEPARATION; CONGENITAL ERYTHROPOIETIC PORPHYRIA; FLOW CYTOMETRY; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HUMAN; LYMPHOCYTE; METABOLISM; RETROVIRUS;

ADULT; AMINOLEVULINIC ACID; CELL LINE; CELL SEPARATION; FLOW CYTOMETRY; GENE THERAPY; GENETIC VECTORS; HUMANS; LYMPHOCYTES; MELATONIN; PORPHYRIA, ERYTHROPOIETIC; PORPHYRINS; RETROVIRIDAE; TRANSDUCTION, GENETIC;

EID: 0033182196     PISSN: 1099498X     EISSN: None     Source Type: Journal    
DOI: 10.1002/(sici)1521-2254(199909/10)1:5<322::aid-jgm53>3.0.co;2-2     Document Type: Article

References (38)

1. Kappas A, Sassa S, Galbraith RA, Nordmann Y. The porphyrias. In The metabolic basis of inherited disease, Scriver CR, Beaudet AL, Sly WS, Valle E (eds). New York: McGravv Hill, 1989; 1305-1365.
2. Romeo G, Levin EY. Uroporphyrinogen III cosynthase in human congenital erythropoietic porphyria. Biochemistry 1969; 63: 856-863.
3. Deybach JC, Verneuil H de, Phung N, et al. Congenital erythropoietic porphyria (Günthers disease): enzymatic studies on two cases of late onset. J Lab Clin Med 1981; 97: 551-558.
4. Verstraeten L, Van Regemorter N, Pardou A, et al. Biochemical diagnosis of a fatal case of Gunther's disease in a newborn with hydrops foetalis. Eur J Clin Chem Clin Biochem 1993; 31: 121-128.
5. Verneuil H de, Moreau-Gaudry F, Ged C, et al. Congenital erythropoietic porphyria. A propos of a fatal case in the neonatal period due to acute hemolysis with hepatic failure. Arch Pediatr 1995; 2: 755-761.
6. Thomas C, Ged C, Nordmann Y, et al. Correction of congenital erythropoietic porphyria by bone marrow transplantation. J Pediatr 1996; 129: 453-456.
7. Moreau-Gaudry F, Mazurier F, Bensidhoum M, Ged C, Verneuil H de. Metabolic correction of congenital erythropoietic porphyria by retrovirus-mediated gene transfer into Epstein-Barr virus-transformed B-cell lines. Blood 1995; 85: 1449-1453.
8. Moreau-Gaudry F, Ged C, Barbot C, et al. Correction of the enzyme defect in cultured congenital erythropoietic porphyria disease cells by retrovirus-mediated gene transfer. Hum Gene Ther 1995; 6: 13-20.
9. Moreau-Gaudry F, Barbot C, Mazurier F, et al. Correction of the enzyme deficit of bone marrow cells in congenital erythropoietic porphyria by retroviral gene transfer. Hematol Cell Ther 1996; 38: 217-220.
10. Mazurier F, Moreau-Gaudry F, Salesse S, et al. Gene transfer of the uroporphyrinogen III synthase cDNA into haematopoietic progenitor cells in view of a future gene therapy in congenital erythropoietic porphyria. J Inherit Metab Dis 1997; 20: 247-257.
11. Nolta A, Kohn D. Haematopoietic stem cells for gene therapy. In Stem cells, Potten CS (ed). London: Academic Press, 1997; 447-462.
12. Princ FG, Juknat AA, Maxit AG, Cardalda C, Batlle A. Melatonin's antioxidant protection against delta-aminolevulinic acid-induced oxidative damage in rat cerebellum. J Pineal Res 1997; 23: 40-46.
13. Tsai SF, Bishop DF, Desnick RJ. Coupled-enzyme and direct assays for uroporphyrinogen III synthase activity in human erythrocytes and cultured lymphoblasts. Anal Biochem 1987; 166: 120-133.
14. Malik Z, Lugaci H. Destruction of erythroleukaemic cells by photoactivation of endogenous porphyrins. Br J Cancer 1987; 56: 589-595.
15. Malik Z, Ehrenberg B, Faraggi A. Inactivation of erythrocytic, lymphocytic and myelocytic leukemic cells by photoexcitation of endogenous porphyrins. J Photochem Photobiol B 1989; 4: 195-205.
16. Vonarx-Coinsman V, Foultier MT, de Brito LX, et al. HepG2 human hepatocarcinoma cells: an experimental model for photosensitization by endogenous porphyrins. J Photochem Photobiol B 1995; 30: 201-208.
17. Fuchs C, Riesenberg R, Siegert J, Baumgartner R. pH-dependent formation of δ-aminolaevulinic acid-induced protoporphyrin IX in fibrosarcoma cells. J Photochem Photobiol B 1997; 40: 49-54.
18. Datta SN, Allman R, Loh CS, Mason M, Matthews PN. Photodynamic therapy of bladder cancer cell lines. Br J Urol 1997; 80: 421-426.
19. Neal R, Yang P, Fiechtl J, et al. Pro-oxidant effects of δ-aminolevulinic acid (δ-ALA) on chinese hamster ovary (CHO) cells. Toxicol Lett 1997; 91: 169-178.
20. Verneuil H de, Ged C, Boulechfar S, Moreau-Gaudry F. Porphyrias: animal models and prospects for cellular and gene therapy. J Bioenerg Biomembr 1995; 27: 239-248.
21. Fontanellas A, Bensidhoum M, Enriquez de Salamanca R, et al. A systematic analysis of the mutations of the uroporphyrinogen III synthase gene in congenital erythropoietic porphyria. Eur J Hum Genet 1996; 4: 274-282.
22. Hermes-Lima M, Valle VGR, Vercesi AE, Bechara EJH. Damage to rat liver mitochondria promoted by delta-aminolevulinic acid-generated reactive oxygen species: connections with acute intermittent porphyria and lead-poisoning. Biochim Biophys Acta 1991; 1056: 57-63.
23. Gilaberte Y, Pereboom D, Carapeto FJ, Aida JO. Flow cytometry study of the role of superoxide anion and hydrogen peroxide in cellular photodestruction with δ-aminolevulinic acid-induced protoporphyrin IX. Photodermatol Photoimmunol Photomed 1997; 13: 43-49.
24. Monteiro HP, Abdalla DS, Augusto O, Bechara EJ. Free radical generation during delta-aminolevulinic acid autoxidation: induction by hemoglobin and connections with porphyrinpathies. Arch Biochem Biophys 1989; 271: 206-216.
25. Van Steveninck J, Boegheim JP, Dubbelman TM, Van der Zee J. The influence of porphyrins on iron-catalysed generation of hydroxyl radicals. Biochem J 1988; 250: 197-201.
26. Koningsberger JC, Rademakers LH, van Hattum J, et al. Exogenous protoporphyrin inhibits Hep G2 cell proliferation, increases the intracellular hydrogen peroxide concentration and causes ultrastructural alterations. J Hepatol 1995; 22: 57-65.
27. Koningsberger JC, Van Asbeck BS, Van Hattum J, et al. The effect of porphyrins on cellular redox systems: a study on the dark effect of porphyrins on phagocytes. Eur J Clin Invest 1993; 23: 716-723.
28. Carneiro RC, Reiter RJ. Delta-aminolevulinic acid-induced lipid peroxidation in rat kidney and liver is attenuated by melatonin: an in vitro and in vivo study. J Pineal Res 1998; 24: 131-136.
29. Princ FG, Maxit AG, Cardalda C, Batlle A, Juknat AA. In vivo protection by melatonin against delta-aminolevulinic acid-induced oxidative damage and its antioxidant effect on the activity of haem enzymes. J Pineal Res 1998; 24: 1-8.
30. Steinbach P, Weingandt H, Baumgartner R, et al. Cellular fluorescence of the endogenous photosensitizer protoporphyrin IX following exposure to delta-aminolevulinic acid. Photochem Photobiol 1995; 62: 887-895.
31. Yeyati PL, Agmon V, Filial C, et al. Fluorescence-based selection of retrovirally transduced cells in the absence of a marker gene: direct selection of transduced type B Niemann-Pick disease cells and evidence for bystander correction. Hum Gene Ther 1995; 6: 975-983.
32. Erlich S, Miranda SR, Visser JW, et al. Fluorescence-based selection of gene-corrected hematopoietic stem and progenitor cells from acid sphingomyelinase-deficient mice: implications for Niemann-Pick disease gene therapy and the development of improved stem cell gene transfer procedures. Blood 1999; 93: 80-86.
33. Peng Q, Berg K, Moan J, Kongshaug M, Neshand JM. δ-Aminolevulinic acid-based photodynamic therapy: principle and experimental research. Photochem Photobiol 1997; 65: 235-251.
34. Navone NM, Polo CF, Frisardi AL, Andrade NE, Batlle C. Heme biosynthesis in human breast cancer-mimetic. In vitro studies and some heme enzymic activity levels. Int J Biochem 1990; 22: 1407-1411.
35. Atzpodien J, Gulati SC, Clarkson BD. Comparison of the cytotoxic effects of merocyanine-540 on leukemic cells and normal human bone marrow. Cancer Res 1986; 46: 4892-4895.
36. Sieber F. Elimination of residual rumor cells from autologous bone marrow grafts by dye-mediated photolysis: preclinical data. Photochem Photobiol 1987; 46: 71-76.
37. Sieber F, Krueger GJ. Photodynamic therapy and bone marrow transplantation. Semin Hematol 1989; 26: 35-39.
38. Eleouet S, Carre J, Vonarx V, et al. Delta-aminolevulinic acid-induced fluorescence in normal human lymphocytes. J Photochem Photobiol B 1997; 41: 22-29.