Therapeutic evaluation of GM2 gangliosidoses by ELISA using anti-GM2 ganglioside antibodies

Clinica Chimica Acta

Volumn 378, Issue 1-2, 2007, Pages 38-41

  Tsuji, Daisuke ^a,b   Higashine, Yukari ^a   Matsuoka, Kazuhiko ^a,b   Sakuraba, Hitoshi ^b,c   Itoh, Kohji ^a,b  

^a Graduate School of Pharmaceutical Sciences   (Japan)

^b JAPAN SCIENCE AND TECHNOLOGY AGENCY   (Japan)

^c TOKYO METROPOLITAN INSTITUTE OF MEDICAL SCIENCE   (Japan)

Author keywords

Cell ELISA; Diagnosis; Enzyme replacement therapy; GM2 ganglioside; Sandhoff disease; Tay Sachs disease

Indexed keywords

GANGLIOSIDE ANTIBODY; GANGLIOSIDE GM2; GANGLIOSIDE GM2 ANTIBODY; UNCLASSIFIED DRUG;

ARTICLE; CONTROLLED STUDY; ENZYME LINKED IMMUNOSORBENT ASSAY; ENZYME REPLACEMENT; FIBROBLAST; GM2 GANGLIOSIDOSIS; HUMAN; HUMAN CELL; LIPID STORAGE; PRIORITY JOURNAL; SANDHOFF DISEASE; TAY SACHS DISEASE;

ANTIBODIES, MONOCLONAL; BETA-N-ACETYLHEXOSAMINIDASE; CELLS, CULTURED; ENZYME-LINKED IMMUNOSORBENT ASSAY; FIBROBLASTS; G(M2) GANGLIOSIDE; GANGLIOSIDOSES, GM2; HUMANS; SANDHOFF DISEASE; TAY-SACHS DISEASE;

EID: 33846937578     PISSN: 00098981     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.cca.2006.10.010     Document Type: Article

References (13)

1. Gravel A., Kaback M., Proia R.L., Sandhoff K., Suzuki K., and Suzuki K. The GM2 gangliosidoses. In: Scriver C.R., Beaudet A.L., Sly W.S., and Valle D. (Eds). The Metabolic and Molecular Bases of Inherited Disease. 8th ed. (2001), McGraw-Hill, New York 3827-3876
2. Mahuran D.J. Biochemical consequences of mutations causing the GM2 gangliosidoses. Biochim Biophys Acta 1455 (1999) 105-138
3. Barton N.W., Brady R.O., Dambrosia J.M., et al. Replacement therapy for inherited enzyme deficiency-macrophage-targeted glucocerebrosidase for Gaucher disease. N Engl J Med 23 (1991) 1464-1470
4. Norflus F., Tifft C.J., McDonald M.P., et al. Bone marrow transplantation prolongs life span and ameliorates neurologic manifestations in Sandhoff disease mice. J Clin Invest 101 (1998) 1881-1888
5. Consiglio A., Quattrini A., Martino S., et al. In vivo gene therapy of metachromatic leukodystrophy by lentiviral vectors: correction of neuropathology and protection against learning impairments in affected mice. Nat Med 7 (2001) 310-316
6. Tsuji D., Kuroki A., Ishibashi Y., Itakura T., and Itoh K. Metabolic correction in microglia derived from Sandhoff disease model mice. J Neurochem 94 (2005) 1631-1638
7. Kotani M., Ozawa H., Kawashima I., Ando S., and Tai T. Generation of one set of monoclonal antibodies specific for a-pathway ganglio-series gangliosides. Biochim Biophys Acta 1117 (1992) 97-103
8. Sakuraba H., Itoh K., Shimmoto M., et al. GM2 gangliosidosis AB variant: clinical and biochemical studies of a Japanese patient. Neurology 52 (1999) 372-377
9. Ohsawa M., Kotani M., Tajima Y., et al. Establishment of immortalized Schwann cells from Sandhoff mice and corrective effect of recombinant human beta-hexosaminidase A on the accumulated GM2 ganglioside. J Hum Genet 50 (2005) 460-467
10. Kornfeld S., and Sly W.S. I-cell disease and pseudo-Hurler polydystrophy: disorders of lysosomal enzyme phosphorylation and localization. In: Scriver C.R., Beaudet A.L., Sly W.S., and Valle D. (Eds). The Metabolic and Molecular Bases of Inherited Disease. 8th ed. (2001), McGraw-Hill, New York 3469-3482
11. Arfi A., Bourgoin C., Basso L., et al. Bicistronic lentiviral vector corrects beta-hexosaminidase deficiency in transduced and cross-corrected human Sandhoff fibroblasts. Neurobiol Dis 20 (2005) 583-593
12. Tsui Z.-C., Chen Q.-R., Thomas M.J., et al. A method for prowling gangliosides in animal tissues using electrospray ionization-tandem mass spectrometry. Anal Biochem 341 (2005) 251-258
13. Tsuji D., Kuroki A., Ishibashi Y., et al. Specific induction of macrophage inflammatory protein 1-alpha in glial cells of Sandhoff disease model mice associated with accumulation of N-acetylhexosaminyl glycoconjugates. J Neurochem 92 (2005) 1497-1507