Masaharu Kotani1, Hideo Yamada, Hitoshi Sakuraba. 1. Department of Clinical Genetics, The Tokyo Metropolitan Institute of Medical Science, Tokyo Metropolitan Organization for Medical Research, 3-18-22 Honkomagome, Bunkyo, Tokyo 113-8613, Japan.
Abstract
BACKGROUND: To clarify the pathogenesis of and evaluate experimental therapeutic trials for lysosomal diseases, effective tools for the detection of intracellularly accumulated materials are required. METHODS: We examined a series of lectins for staining and blotting of the accumulated glycoconjugates in sialidosis and galactosialidosis. RESULTS: Lysosomally accumulated sialyl glycoconjugates were successfully detected in cultured fibroblasts from patients with these diseases by means of staining and blotting with Macckia amurensis (MAM). CONCLUSIONS: This procedure is sensitive and easy, and will be useful not only for biochemical and diagnostic analyses, but also for therapeutic evaluation in these diseases. Copyright 2004 Elsevier B.V.
BACKGROUND: To clarify the pathogenesis of and evaluate experimental therapeutic trials for lysosomal diseases, effective tools for the detection of intracellularly accumulated materials are required. METHODS: We examined a series of lectins for staining and blotting of the accumulated glycoconjugates in sialidosis and galactosialidosis. RESULTS: Lysosomally accumulated sialyl glycoconjugates were successfully detected in cultured fibroblasts from patients with these diseases by means of staining and blotting with Macckia amurensis (MAM). CONCLUSIONS: This procedure is sensitive and easy, and will be useful not only for biochemical and diagnostic analyses, but also for therapeutic evaluation in these diseases. Copyright 2004 Elsevier B.V.