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

GM2 gangliosidoses, including Tay-Sachs disease, Sandhoff disease and the AB variant, comprise deficiencies of β-hexosaminidase isozymes and GM2 ganglioside activator protein associated with accumulation of GM2 ganglioside (GM2) in lysosomes and neurosomatic clinical manifestations. A simple assay s...

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Veröffentlicht in:Clinica chimica acta 2007-03, Vol.378 (1), p.38-41
Hauptverfasser: Tsuji, Daisuke, Higashine, Yukari, Matsuoka, Kazuhiko, Sakuraba, Hitoshi, Itoh, Kohji
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container_issue 1
container_start_page 38
container_title Clinica chimica acta
container_volume 378
creator Tsuji, Daisuke
Higashine, Yukari
Matsuoka, Kazuhiko
Sakuraba, Hitoshi
Itoh, Kohji
description GM2 gangliosidoses, including Tay-Sachs disease, Sandhoff disease and the AB variant, comprise deficiencies of β-hexosaminidase isozymes and GM2 ganglioside activator protein associated with accumulation of GM2 ganglioside (GM2) in lysosomes and neurosomatic clinical manifestations. A simple assay system for intracellular quantification of GM2 is required to evaluate the therapeutic effects on GM2-gangliosidoses. We newly established a cell–ELISA system involving anti-GM2 monoclonal antibodies for measuring GM2 storage in fibroblasts from Tay-Sachs and Sandhoff disease patients. We succeeded in detecting the corrective effect of enzyme replacement on elimination of GM2 in the cells with this ELISA system. This simple and sensitive system should be useful as additional diagnosis tool as well as therapeutic evaluation of GM2 gangliosidoses.
doi_str_mv 10.1016/j.cca.2006.10.010
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subjects Antibodies, Monoclonal - therapeutic use
beta-Hexosaminidase beta Chain
beta-N-Acetylhexosaminidases - therapeutic use
Cells, Cultured
Cell–ELISA
Diagnosis
Enzyme replacement therapy
Enzyme-Linked Immunosorbent Assay - methods
Fibroblasts - chemistry
G(M2) Ganglioside - analysis
G(M2) Ganglioside - immunology
Gangliosidoses, GM2 - therapy
GM2 ganglioside
Hexosaminidase B
Humans
Sandhoff disease
Sandhoff Disease - therapy
Tay-Sachs disease
Tay-Sachs Disease - therapy
title Therapeutic evaluation of GM2 gangliosidoses by ELISA using anti-GM2 ganglioside antibodies
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