{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,1]],"date-time":"2025-11-01T13:47:15Z","timestamp":1762004835767},"reference-count":47,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2016,10,14]],"date-time":"2016-10-14T00:00:00Z","timestamp":1476403200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"},{"start":{"date-parts":[[2016,10,14]],"date-time":"2016-10-14T00:00:00Z","timestamp":1476403200000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J of Inher Metab Disea"],"published-print":{"date-parts":[[2017,3]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Mucopolysaccharidosis type I (MPS IH) is a lysosomal storage disease (LSD) caused by inactivating mutations to the alpha\u2010<jats:italic>L<\/jats:italic>\u2010iduronidase (IDUA) gene. Treatment focuses on IDUA enzyme replacement and currently employed methods can be non\u2010uniform in their efficacy particularly for the cardiac and craniofacial pathology. Therefore, we undertook efforts to better define the pathological cascade accounting for treatment refractory manifestations and demonstrate a role for the renin angiotensin system (RAS) using the IDUA<jats:sup>\u2212\/\u2212<\/jats:sup> mouse model. Perturbation of the RAS in the aorta was more profound in male animals suggesting a causative role in the observed gender dimorphism and angiotensin receptor blockade (ARB) resulted in improved cardiac function. Further, we show the ability of losartan to prevent shortening of the snout, a common craniofacial anomaly in IDUA<jats:sup>\u2212\/\u2212<\/jats:sup> mice. These data show a key role for the RAS in MPS associated pathology and support the inclusion of losartan as an augmentation to current therapies.<\/jats:p>","DOI":"10.1007\/s10545-016-9988-z","type":"journal-article","created":{"date-parts":[[2016,10,14]],"date-time":"2016-10-14T12:21:11Z","timestamp":1476447671000},"page":"281-289","update-policy":"http:\/\/dx.doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":12,"title":["Angiotensin receptor blockade mediated amelioration of mucopolysaccharidosis type I cardiac and craniofacial pathology"],"prefix":"10.1002","volume":"40","author":[{"given":"Mark J.","family":"Osborn","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Division of Blood and Marrow Transplantation University of Minnesota 420 Delaware ST SE, MMC 366 Minneapolis MN 55455 USA"},{"name":"Center for Genome Engineering University of Minnesota Minneapolis MN USA"},{"name":"Stem Cell Institute University of Minnesota Minneapolis MN USA"},{"name":"Asan\u2010Minnesota Institute for Innovating Transplantation Seoul Republic of Korea"},{"name":"School of Public Health University of Minnesota Minneapolis MN USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Beau R.","family":"Webber","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Blood and Marrow Transplantation University of Minnesota 420 Delaware ST SE, MMC 366 Minneapolis MN 55455 USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ronald T.","family":"McElmurry","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Blood and Marrow Transplantation University of Minnesota 420 Delaware ST SE, MMC 366 Minneapolis MN 55455 USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kyle D.","family":"Rudser","sequence":"additional","affiliation":[{"name":"Department of Cell Biology and Anatomy and the Alberta Children's Hospital Research Institute University of Calgary Calgary AB Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anthony P.","family":"DeFeo","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Blood and Marrow Transplantation University of Minnesota 420 Delaware ST SE, MMC 366 Minneapolis MN 55455 USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michael","family":"Muradian","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Blood and Marrow Transplantation University of Minnesota 420 Delaware ST SE, MMC 366 Minneapolis MN 55455 USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anna","family":"Petryk","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Blood and Marrow Transplantation University of Minnesota 420 Delaware ST SE, MMC 366 Minneapolis MN 55455 USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Benedikt","family":"Hallgrimsson","sequence":"additional","affiliation":[{"name":"Department of Cell Biology and Anatomy and the Alberta Children's Hospital Research Institute University of Calgary Calgary AB Canada"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bruce R.","family":"Blazar","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Blood and Marrow Transplantation University of Minnesota 420 Delaware ST SE, MMC 366 Minneapolis MN 55455 USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jakub","family":"Tolar","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Blood and Marrow Transplantation University of Minnesota 420 Delaware ST SE, MMC 366 Minneapolis MN 55455 USA"},{"name":"Stem Cell Institute University of Minnesota Minneapolis MN USA"},{"name":"Asan\u2010Minnesota Institute for Innovating Transplantation Seoul Republic of Korea"},{"name":"School of Public Health University of Minnesota Minneapolis MN USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Elizabeth A.","family":"Braunlin","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Blood and Marrow Transplantation University of Minnesota 420 Delaware ST SE, MMC 366 Minneapolis MN 55455 USA"},{"name":"Lillehei Heart Institute University of Minnesota Minneapolis MN USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2016,10,14]]},"reference":[{"key":"e_1_2_9_2_1","doi-asserted-by":"publisher","DOI":"10.1152\/ajprenal.00145.2002"},{"key":"e_1_2_9_3_1","first-page":"PE16","article-title":"Angiotensin type 2 receptor (AT2R): a challenging twin","volume":"2003","author":"Berk BC","year":"2003","journal-title":"Scie STKE: Sig Trans Knowl Environ"},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1203\/01.pdr.0000190579.24054.39"},{"key":"e_1_2_9_5_1","doi-asserted-by":"publisher","DOI":"10.1113\/expphysiol.2007.041806"},{"key":"e_1_2_9_6_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2015.02.006"},{"key":"e_1_2_9_7_1","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCULATIONAHA.111.066712"},{"key":"e_1_2_9_8_1","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/6.4.503"},{"key":"e_1_2_9_9_1","first-page":"1329","article-title":"Abnormal muscle mechanosignaling triggers cardiomyopathy in mice with Marfan syndrome","volume":"124","author":"Cook JR","year":"2014","journal-title":"J Clin Invest"},{"key":"e_1_2_9_10_1","doi-asserted-by":"publisher","DOI":"10.1161\/01.HYP.35.6.1183"},{"key":"e_1_2_9_11_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.peptides.2011.06.018"},{"key":"e_1_2_9_12_1","doi-asserted-by":"publisher","DOI":"10.1161\/01.HYP.28.6.1055"},{"key":"e_1_2_9_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.4780111304"},{"key":"e_1_2_9_14_1","doi-asserted-by":"publisher","DOI":"10.1542\/peds.2009-1728"},{"key":"e_1_2_9_15_1","doi-asserted-by":"publisher","DOI":"10.1152\/ajpheart.00426.2008"},{"key":"e_1_2_9_16_1","doi-asserted-by":"publisher","DOI":"10.1126\/science.1192152"},{"key":"e_1_2_9_17_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11692-009-9076-5"},{"key":"e_1_2_9_18_1","first-page":"561","volume-title":"Current topics in developmental biology","author":"Hallgrimsson B","year":"2015"},{"key":"e_1_2_9_19_1","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.4199-14.2015"},{"key":"e_1_2_9_20_1","doi-asserted-by":"publisher","DOI":"10.1002\/jgm.1229"},{"key":"e_1_2_9_21_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11906-012-0319-y"},{"key":"e_1_2_9_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/jcp.10299"},{"key":"e_1_2_9_23_1","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCULATIONAHA.105.000810"},{"key":"e_1_2_9_24_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1755-0998.2010.02924.x"},{"key":"e_1_2_9_25_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymthe.2004.08.027"},{"key":"e_1_2_9_26_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1440-1746.2008.05461.x"},{"key":"e_1_2_9_27_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.ymgme.2008.03.018"},{"key":"e_1_2_9_28_1","doi-asserted-by":"publisher","DOI":"10.1006\/jmcc.1997.0489"},{"key":"e_1_2_9_29_1","doi-asserted-by":"publisher","DOI":"10.1177\/1753944715597623"},{"key":"e_1_2_9_30_1","doi-asserted-by":"publisher","DOI":"10.1161\/CIRCRESAHA.111.253740"},{"key":"e_1_2_9_31_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2009.236"},{"key":"e_1_2_9_32_1","doi-asserted-by":"publisher","DOI":"10.1007\/s11692-009-9055-x"},{"key":"e_1_2_9_33_1","volume-title":"The metabolic and molecular bases of inherited disease","author":"Neufeld EF","year":"2001"},{"key":"e_1_2_9_34_1","doi-asserted-by":"publisher","DOI":"10.1038\/mt.2010.249"},{"key":"e_1_2_9_35_1","doi-asserted-by":"publisher","DOI":"10.1111\/joa.12382"},{"key":"e_1_2_9_36_1","doi-asserted-by":"publisher","DOI":"10.1172\/JCI46215"},{"key":"e_1_2_9_37_1","doi-asserted-by":"publisher","DOI":"10.1093\/cvr\/cvs263"},{"key":"e_1_2_9_38_1","doi-asserted-by":"publisher","DOI":"10.1002\/sim.2682"},{"key":"e_1_2_9_39_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.amjhyper.2004.09.009"},{"key":"e_1_2_9_40_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.peptides.2009.09.005"},{"key":"e_1_2_9_41_1","doi-asserted-by":"publisher","DOI":"10.1681\/ASN.V12122609"},{"key":"e_1_2_9_42_1","doi-asserted-by":"publisher","DOI":"10.1177\/1074248410381757"},{"key":"e_1_2_9_43_1","first-page":"524","article-title":"Gender\u2010related dimorphism in aortic insufficiency in murine mucopolysaccharidosis type I","volume":"18","author":"Tolar J","year":"2009","journal-title":"J Heart Valve Dis"},{"key":"e_1_2_9_44_1","doi-asserted-by":"publisher","DOI":"10.1097\/HJH.0b013e3282f465d2"},{"key":"e_1_2_9_45_1","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.273.46.30795"},{"key":"e_1_2_9_46_1","doi-asserted-by":"publisher","DOI":"10.2215\/CJN.06620909"},{"key":"e_1_2_9_47_1","doi-asserted-by":"publisher","DOI":"10.4077\/CJP.2014.BAD268"},{"key":"e_1_2_9_48_1","doi-asserted-by":"publisher","DOI":"10.1152\/ajpheart.00814.2014"}],"container-title":["Journal of Inherited Metabolic Disease"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s10545-016-9988-z.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/article\/10.1007\/s10545-016-9988-z\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"http:\/\/link.springer.com\/content\/pdf\/10.1007\/s10545-016-9988-z.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1007\/s10545-016-9988-z","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T08:48:56Z","timestamp":1694767736000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1007\/s10545-016-9988-z"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2016,10,14]]},"references-count":47,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2017,3]]}},"alternative-id":["10.1007\/s10545-016-9988-z"],"URL":"https:\/\/doi.org\/10.1007\/s10545-016-9988-z","archive":["Portico"],"relation":{},"ISSN":["0141-8955","1573-2665"],"issn-type":[{"value":"0141-8955","type":"print"},{"value":"1573-2665","type":"electronic"}],"subject":[],"published":{"date-parts":[[2016,10,14]]}}}