Recombinant encapsulated cells overexpressing alpha-L-iduronidase correct enzyme deficiency in human mucopolysaccharidosis type i cells

Cells Tissues Organs

Volumn 195, Issue 4, 2012, Pages 323-329

  Baldo, Guilherme ^a   Quoos Mayer, Fabiana ^a   Burin, Maira ^b   Carrillo Farga, Joaqun ^c   Matte, Ursula ^a   Giugliani, Roberto ^a  

^a FEDERAL UNIVERSITY OF RIO GRANDE DO SUL   (Brazil)

^b HOSPITAL DE CLÍNICAS DE PORTO ALEGRE   (Brazil)

^c Institute of Hemopathology   (Mexico)

Author keywords

Cell encapsulation; Cryopreservation; Gene therapy; Mannose 6 phosphate receptor; Mucopolysaccharidosis type I

Indexed keywords

COMPLEMENTARY DNA; GLYCOSAMINOGLYCAN; LEVO IDURONIDASE; MANNOSE 6 PHOSPHATE; RECOMBINANT ENZYME; SOMATOMEDIN B RECEPTOR;

ANIMAL CELL; ARTICLE; CELL COUNT; CELL ENCAPSULATION; CELL GROWTH; CELL STRAIN BHK; CELL VIABILITY; COCULTURE; CONCENTRATION RESPONSE; CONTROLLED STUDY; CRYOPRESERVATION; CYTOLOGY; ENZYME ACTIVITY; ENZYME RELEASE; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; HURLER SYNDROME; INTERNALIZATION; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION; SKIN FIBROBLAST;

ANIMALS; CAPSULES; CELL LINE; CELL PROLIFERATION; CELL SURVIVAL; CELLS, IMMOBILIZED; COCULTURE TECHNIQUES; CRICETINAE; CRYOPRESERVATION; FIBROBLASTS; GLYCOSAMINOGLYCANS; HUMANS; IDURONIDASE; MUCOPOLYSACCHARIDOSIS I; RECEPTOR, IGF TYPE 2; RECOMBINATION, GENETIC;

CRICETINAE;

EID: 84859004695     PISSN: 14226405     EISSN: 14226421     Source Type: Journal    
DOI: 10.1159/000327532     Document Type: Article

References (18)

1. Bloch, J., A.C. Bachoud-Lévi, N. Déglon, J.P. Lefaucheur, L. Winkel, S. Palfi, J.P. Nguyen, C. Bourdet, V. Gaura, P. Remy, P. Brugires, M.F. Boisse, S. Baudic, P. Cesaro, P. Hantraye, P. Aebischer, M. Peschanski (2004) Neuroprotective gene therapy for Huntingtons disease, using polymer-encapsulated cells engineered to secrete human ciliary neurotrophic factor: results of a phase I study. Hum Gene Ther 15: 968-975. (Pubitemid 39372573)
2. Bressel, T., A.H. Paz, G. Baldo, E.O. Cirne-Lima, U. Matte, M.L.S. Pereira (2008) An effective device for generating alginate microcapsules. Genet Mol Biol 31: 136-140. (Pubitemid 351780661)
3. Carrillo-Farga, J., M.A. Acosta-Barreda, M.E. Gomez-Ruiz, E. Monterrubio, R. Pérez-Landa (1997) El diagnóstico de enfermedades lisosomales hereditarias por morfología de células hematopoyéticas. Rev Mex Patol Clin 44: 222-232.
4. Charp, P.A., R.J. Kinders, T.C. Johnson (1983) G2 cell cycle arrest induced by glycopeptides isolated from the bovine cerebral cortex. J Cell Biol 97: 311-316.
5. Hopwood, J.J., V. Muller, A. Smithson, N. Baggett (1979) A fluorometric assay using 4-methylumbelliferyl alpha-L -iduronide for the estimation of alpha-L -iduronidase activity and the detection of Hurler and Scheie syndromes. Clin Chim Acta 92: 257-265. (Pubitemid 9148277)
6. Lagranha, V.L., G. Baldo, T.G. de Carvalho, M. Burin, M.L. Saraiva-Pereira, U. Matte, R. Giu gliani (2008) In vitro correction of ARSA deficiency in human skin fibroblasts from metachromatic leukodystrophy patients after treatment with microencapsulated recombinant cells. Metab Brain Dis 23: 469-484.
7. Lange, M.C., H.A. Teive, A.R. Troiano, M. Bitencourt, V.A. Funke, D.C. Setúbal, J. Zanis Neto, C.R. Medeiros, L.C. Werneck, R. Pasquini, C.M. Bonfim (2006) Bone marrow transplantation in patients with storage diseases: a developing country experience. Arq Neuropsiquiatr 64: 1-4.
8. Ma, X., M. Tittiger, R.H. Knutsen, A. Kovacs, L. Schaller, R.P. Mecham, K.P. Ponder (2008) Upregulation of elastase proteins results in aortic dilatation in mucopolysaccharidosis I mice. Mol Genet Metab 94: 298-304.
9. Martins, A.M., A.P. Dualibi, D. Norato, E.T. Takata, E.S. Santos, E.R. Valadares, G. Porta, G. de Luca, G. Moreira, H. Pimentel, J. Coelho, J.M. Brum, J. Semionato Filho, M.S. Kers tenetzky, M.R. Guimarães, M.V. Rojas, P.C. Aranda, R.F. Pires, R.G. Faria, R.M. Mota, U. Matte, Z.C. Guedes (2009) Guidelines for the management of mucopolysaccharidosis type I. J Pediatr 155: S32-S46.
10. Mayer, F.Q., G. Baldo, T.G. de Carvalho, V.L. Lagranha, R. Giugliani, U. Matte (2010) Effects of cryopreservation and hypothermic storage on cell viability and enzyme activity in recombinant encapsulated cells overexpressing alpha-L -iduronidase. Artif Organs 34: 434-439.
11. Munoz-Rojas, M.V., T. Vieira, R. Costa, S. Fagondes, A. John, L.B. Jardim, L.M. Vedolin, M. Raymundo, P.I. Dickson, E. Kakkis, R. Giugliani (2008) Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression. Am J Med Genet A 146A: 2538-2544.
12. Nakama, H., K. Ohsugi, T. Otsuki, I. Date, M. Kosuga, T. Okuyama, N. Sakuragawa (2006) Encapsulation cell therapy for mucopolysaccharidosis type VII using genetically engineered immortalized human amniotic epithelial cells. Tohoku J Exp Med 209: 23-32.
13. Orive, G., R.M. Hernndez, A. Rodríguez Gascón, R. Calafiore, T.M. Chang, P. de Vos, G. Hortelano, D. Hunkeler, I. Lacík, J.L. Pedraz (2004) History, challenges and perspectives of cell microencapsulation. Trends Biotechnol 22: 87-92. (Pubitemid 38130176)
14. Sands, M.S., C.A. Vogler, K.K. Ohlemiller, M.S. Roberts, J.H. Grubb, B. Levy, W.S. Sly (2001) Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII. J Biol Chem 276: 43160-43165.
15. Sly, W.S., C. Vogler, J.H. Grubb, B. Levy, N. Galvin, Y. Tan, T. Nishioka, S. Tomatsu (2006) Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors. Proc Natl Acad Sci USA 103: 15172-15177. (Pubitemid 44583183)
16. Tsukimura, T., Y. Tajima, I. Kawashima, T. Fukushige, T. Kanzaki, T. Kanekura, M. Ikekita, K. Sugawara, T. Suzuki, T. Togawa, H. Sakuraba (2008) Uptake of a recombinant human alpha-L -iduronidase (laronidase) by cultured fibroblasts and osteoblasts. Biol Pharm Bull 31: 1691-1695.
17. Wraith, J.E. (2009) Enzyme replacement therapy for the management of the mucopolysaccharidoses. Int J Clin Pharmacol Ther 47: S63-S65.
18. Yang, R.Y., D.K. Hsu, F.T. Liu (1996) Expression of galectin-3 modulates T-cell growth and apoptosis. Proc Natl Acad Sci USA 93: 6737-6742. (Pubitemid 26230379)