{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T01:36:52Z","timestamp":1790473012613,"version":"4.1.0"},"reference-count":35,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2010,1,19]],"date-time":"2010-01-19T00:00:00Z","timestamp":1263859200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2010,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>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\u2010glucuronidase 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.<\/jats:p>","DOI":"10.1007\/s10545-009-9029-2","type":"journal-article","created":{"date-parts":[[2010,1,18]],"date-time":"2010-01-18T12:42:35Z","timestamp":1263818555000},"page":"61-67","source":"Crossref","is-referenced-by-count":48,"title":["Storage correction in cells of patients suffering from mucopolysaccharidoses types IIIA and VII after treatment with genistein and other isoflavones"],"prefix":"10.1002","volume":"33","author":[{"given":"Audrey","family":"Arfi","sequence":"first","affiliation":[{"name":"Inserm, U640 Paris 75006 France"},{"name":"CNRS, UMR8151 Paris 75006 France"},{"name":"Facult\u00e9 de Pharmacie, Chemical and Genetic Pharmacology Laboratory Universit\u00e9 Paris Descartes Paris 75270 France"},{"name":"Ecole Nationale Sup\u00e9rieure de Chimie de Paris Paris 75005 France"},{"name":"CNRS UMR 8151, Inserm, U640, Facult\u00e9 de Pharmacie 4, avenue de l'Observatoire Paris 75006 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Magali","family":"Richard","sequence":"additional","affiliation":[{"name":"Inserm, U640 Paris 75006 France"},{"name":"CNRS, UMR8151 Paris 75006 France"},{"name":"Facult\u00e9 de Pharmacie, Chemical and Genetic Pharmacology Laboratory Universit\u00e9 Paris Descartes Paris 75270 France"},{"name":"Ecole Nationale Sup\u00e9rieure de Chimie de Paris Paris 75005 France"},{"name":"CNRS UMR 8151, Inserm, U640, Facult\u00e9 de Pharmacie 4, avenue de l'Observatoire Paris 75006 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christelle","family":"Gandolphe","sequence":"additional","affiliation":[{"name":"Inserm, U640 Paris 75006 France"},{"name":"CNRS, UMR8151 Paris 75006 France"},{"name":"Facult\u00e9 de Pharmacie, Chemical and Genetic Pharmacology Laboratory Universit\u00e9 Paris Descartes Paris 75270 France"},{"name":"Ecole Nationale Sup\u00e9rieure de Chimie de Paris Paris 75005 France"},{"name":"CNRS UMR 8151, Inserm, U640, Facult\u00e9 de Pharmacie 4, avenue de l'Observatoire Paris 75006 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Daniel","family":"Scherman","sequence":"additional","affiliation":[{"name":"Inserm, U640 Paris 75006 France"},{"name":"CNRS, UMR8151 Paris 75006 France"},{"name":"Facult\u00e9 de Pharmacie, Chemical and Genetic Pharmacology Laboratory Universit\u00e9 Paris Descartes Paris 75270 France"},{"name":"Ecole Nationale Sup\u00e9rieure de Chimie de Paris Paris 75005 France"},{"name":"CNRS UMR 8151, Inserm, U640, Facult\u00e9 de Pharmacie 4, avenue de l'Observatoire Paris 75006 France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2010,1,19]]},"reference":[{"key":"e_1_2_6_2_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00439-006-0280-4"},{"key":"e_1_2_6_3_1","doi-asserted-by":"publisher","DOI":"10.1093\/ajcn\/76.5.1126"},{"key":"e_1_2_6_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.molmed.2006.06.001"},{"key":"e_1_2_6_5_1","doi-asserted-by":"publisher","DOI":"10.1093\/ajcn\/75.1.126"},{"key":"e_1_2_6_6_1","doi-asserted-by":"publisher","DOI":"10.1136\/adc.69.3.403"},{"key":"e_1_2_6_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1651-2227.2005.tb02116.x"},{"key":"e_1_2_6_8_1","doi-asserted-by":"publisher","DOI":"10.1207\/s15327914nc4802_5"},{"key":"e_1_2_6_9_1","doi-asserted-by":"publisher","DOI":"10.1093\/jn\/136.1.75"},{"issue":"4","key":"e_1_2_6_10_1","doi-asserted-by":"crossref","first-page":"533","DOI":"10.1016\/j.jpeds.2005.12.014","article-title":"Enzyme replacement therapy for mucopolysaccharidosis VI: a phase 3, randomized, double\u2010blind, placebo\u2010controlled, multinational study of recombinant human N\u2010acetylgalactosamine 4\u2010sulfatase (recombinant human arylsulfatase B or rhASB) and follow\u2010on, open\u2010label extension study","volume":"148","author":"Harmatz P, Giugliani R, Schwartz I, et al, MPS VI Phase 3 Study Group","year":"2006","journal-title":"J Pediatr"},{"key":"e_1_2_6_11_1","doi-asserted-by":"publisher","DOI":"10.1007\/BF03195237"},{"key":"e_1_2_6_12_1","doi-asserted-by":"publisher","DOI":"10.1186\/1423-0127-16-26"},{"key":"e_1_2_6_13_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM200101183440304"},{"key":"e_1_2_6_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2009.06.013"},{"key":"e_1_2_6_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.fct.2005.06.004"},{"key":"e_1_2_6_16_1","doi-asserted-by":"publisher","DOI":"10.1093\/ajcn\/77.4.875"},{"key":"e_1_2_6_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1651-2227.2002.tb03118.x"},{"key":"e_1_2_6_18_1","doi-asserted-by":"publisher","DOI":"10.1097\/01.gim.0000232477.37660.fb"},{"key":"e_1_2_6_19_1","first-page":"3421","volume-title":"The metabolic and molecular bases of inherited disease","author":"Neufeld E","year":"2001"},{"key":"e_1_2_6_20_1","doi-asserted-by":"publisher","DOI":"10.1093\/ajcn\/74.4.418"},{"key":"e_1_2_6_21_1","doi-asserted-by":"publisher","DOI":"10.2165\/00126839-200607060-00003"},{"key":"e_1_2_6_22_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.M104677200"},{"key":"e_1_2_6_23_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.ejhg.5201623"},{"key":"e_1_2_6_24_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.curtheres.2008.04.002"},{"key":"e_1_2_6_25_1","doi-asserted-by":"crossref","unstructured":"PiotrowskaE Jak\u00f3bkiewicz\u2010BaneckaJ WegrzynG(2009)Different amounts of isoflavones in various commercially available soy extracts in the light of gene expression\u2010targeted isoflavone therapy.Phytother Res[Epub ahead of print].","DOI":"10.1002\/ptr.2944"},{"key":"e_1_2_6_26_1","doi-asserted-by":"publisher","DOI":"10.1038\/nmeth807"},{"key":"e_1_2_6_27_1","doi-asserted-by":"publisher","DOI":"10.1002\/jnr.21779"},{"key":"e_1_2_6_28_1","doi-asserted-by":"publisher","DOI":"10.1038\/gt.2009.36"},{"key":"e_1_2_6_29_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddm289"},{"key":"e_1_2_6_30_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0022-3476(73)80162-3"},{"key":"e_1_2_6_31_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/ddm353"},{"key":"e_1_2_6_32_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.chroma.2004.10.048"},{"key":"e_1_2_6_33_1","doi-asserted-by":"publisher","DOI":"10.1055\/s-2005-864186"},{"key":"e_1_2_6_34_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10545-008-0838-5"},{"key":"e_1_2_6_35_1","doi-asserted-by":"publisher","DOI":"10.1517\/14656566.6.3.489"},{"key":"e_1_2_6_36_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jpeds.2004.01.046"}],"container-title":["Journal of Inherited Metabolic Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1007\/s10545-009-9029-2","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1007\/s10545-009-9029-2","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1007\/s10545-009-9029-2","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T14:23:12Z","timestamp":1694787792000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1007\/s10545-009-9029-2"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2010,1,19]]},"references-count":35,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2010,2]]}},"alternative-id":["10.1007\/s10545-009-9029-2"],"URL":"https:\/\/doi.org\/10.1007\/s10545-009-9029-2","archive":["Portico"],"relation":{},"ISSN":["0141-8955","1573-2665"],"issn-type":[{"value":"0141-8955","type":"print"},{"value":"1573-2665","type":"electronic"}],"subject":[],"published":{"date-parts":[[2010,1,19]]}}}