Therapeutic evaluation of GM2 gangliosidoses by ELISA using anti-GM2 ganglioside antibodies
D Tsuji, Y Higashine, K Matsuoka, H Sakuraba… - Clinica chimica acta, 2007 - Elsevier
D Tsuji, Y Higashine, K Matsuoka, H Sakuraba, K Itoh
Clinica chimica acta, 2007•ElsevierBACKGROUND: 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. METHODS: We
newly established a cell–ELISA system involving anti-GM2 monoclonal antibodies for …
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. METHODS: We
newly established a cell–ELISA system involving anti-GM2 monoclonal antibodies for …
BACKGROUND
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.
METHODS
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.
RESULTS
We succeeded in detecting the corrective effect of enzyme replacement on elimination of GM2 in the cells with this ELISA system.
CONCLUSIONS
This simple and sensitive system should be useful as additional diagnosis tool as well as therapeutic evaluation of GM2 gangliosidoses.
Elsevier