ArticleJournal of inherited metabolic disease2010
Storage correction in cells of patients suffering from mucopolysaccharidoses types IIIA and VII after treatment with genistein and other isoflavones.
Article in Journal of inherited metabolic disease, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 21 papers.
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Who cites it
21 citing papers in PubMed, 48 citations in OpenAlex.
- Genistein supplementation in patients affected by Sanfilippo disease.Journal of inherited metabolic disease · 2011Trial
- Advances in Therapies for Mucopolysaccharidoses.Current issues in molecular biology · 2026Review
- The use of genistein and ambroxol may be an effective approach in correcting cellular dysfunctions of mucopolysaccharidosis-plus syndrome.Mammalian genome : official journal of the International Mammalian Genome Society · 2025Article
- Mucopolysaccharidoses-What Clinicians Need to Know: A Clinical, Biochemical, and Molecular Overview.Biomolecules · 2025Review
- Article
- Activities of (Poly)phenolic Antioxidants and Other Natural Autophagy Modulators in the Treatment of Sanfilippo Disease: Remarkable Efficacy of Resveratrol in Cellular and Animal Models.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2023Article
- Murine Fibroblasts and Primary Hepatocytes as Tools When Studying the Efficacy of Potential Therapies for Mucopolysaccharidosis Type I.International journal of molecular sciences · 2022Article
- Article
- Cellular and Gene Expression Response to the Combination of Genistein and Kaempferol in the Treatment of Mucopolysaccharidosis Type I.International journal of molecular sciences · 2022Article
- Novel therapies for mucopolysaccharidosis type III.Journal of inherited metabolic disease · 2021Review
- Improved Pharmacokinetics and Tissue Uptake of Complexed Daidzein in Rats.Pharmaceutics · 2020Article
- New therapeutic approaches for Krabbe disease: The potential of pharmacological chaperones.Journal of neuroscience research · 2016Review
- Activities of genes controlling sphingolipid metabolism in human fibroblasts treated with flavonoids.Metabolic brain disease · 2015Article
- RTB Lectin: a novel receptor-independent delivery system for lysosomal enzyme replacement therapies.Scientific reports · 2015Article
- Modulation of expression of genes involved in glycosaminoglycan metabolism and lysosome biogenesis by flavonoids.Scientific reports · 2015Article
- Genistein increases glycosaminoglycan levels in mucopolysaccharidosis type I cell models.Journal of inherited metabolic disease · 2014Article
- The phytoestrogen genistein modulates lysosomal metabolism and transcription factor EB (TFEB) activation.The Journal of biological chemistry · 2014Article
- Synthetic genistein derivatives as modulators of glycosaminoglycan storage.Journal of translational medicine · 2012Article
- The use of elevated doses of genistein-rich soy extract in the gene expression-targeted isoflavone therapy for Sanfilippo disease patients.JIMD reports · 2012Article
- Emerging therapies for neurodegenerative lysosomal storage disorders - from concept to reality.Journal of inherited metabolic disease · 2011Review
Corrections and comments
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Authors and funding
4 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Mucopolysaccharidoses are autosomal and recessive lysosomal storage disorders caused by the deficiency of a lysosomal enzyme involved in glycosaminoglycan catabolism. The Sanfilippo type A disease (MPS III A) results from sulfamidase deficiency, which leads to accumulation of heparan sulfate, whereas Sly disease (MPS VII) results from beta-glucuronidase deficiency, leading to accumulation of heparan, dermatan, and chondroitin sulfates. These syndromes are characterized by severe central nervous system degeneration, resulting in progressive mental retardation, and fatality occurs in severely affected children. To date, no effective treatment is available except for bone marrow transplantation in specific cases. Recently, the use of genistein, an isoflavone that inhibits glycosaminoglycans synthesis, has been tested as substrate reduction therapy for neuronopathic forms of these diseases.We tested five natural analogs to genistein in human fibroblasts from both Sanfilippo A and Sly patients. Four molecules were as efficient as genistein in decreasing glycosaminoglycan accumulation. Moreover, a combination of several isoflavones was more efficient than one single isoflavone, suggesting a synergistic effect. These preliminary data may offer new perspectives for treating Sly and Sanfilippo A diseases and could be relevant to other neurological forms of mucopolysaccharidoses.
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