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Natl. Acad. Sci. U.S.A."],"published-print":{"date-parts":[[2020,4,21]]},"abstract":"<jats:title>Significance<\/jats:title>\n                  <jats:p>Lysosomal storage disorders (LSDs) are one of the most prevalent inherited pediatric conditions. Although some LSDs were described over a century ago, we understand little about how specific cells contribute to pathogenesis. Most LSDs are caused by a lysosomal enzyme deficiency. Lysosomal enzymes can be secreted from one cell and correct neighboring cells (cross-correction). Cross-correction prevents cell-limited expression or deletion of lysosomal enzymes, making it impossible to determine the role of specific cells in LSDs. Here we describe a method to eliminate cross-correction while maintaining enzymatic function. We used this method to determine the role of specific cells (myelin-producing) in Krabbe disease. This same method could be used to determine the role of specific cells in most other LSDs.<\/jats:p>","DOI":"10.1073\/pnas.1917675117","type":"journal-article","created":{"date-parts":[[2020,4,6]],"date-time":"2020-04-06T20:25:58Z","timestamp":1586204758000},"page":"9032-9041","update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":18,"title":["Cell-autonomous expression of the acid hydrolase galactocerebrosidase"],"prefix":"10.1073","volume":"117","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8219-5416","authenticated-orcid":false,"given":"Christina R.","family":"Mikulka","sequence":"first","affiliation":[{"name":"Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-0335-1087","authenticated-orcid":false,"given":"Joshua T.","family":"Dearborn","sequence":"additional","affiliation":[{"name":"Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bruno A.","family":"Benitez","sequence":"additional","affiliation":[{"name":"Department of Psychiatry, Washington University School of Medicine, St. Louis, MO 63110;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy","family":"Strickland","sequence":"additional","affiliation":[{"name":"Department of Genetics, Washington University School of Medicine, St. Louis, MO 63110"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lin","family":"Liu","sequence":"additional","affiliation":[{"name":"Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Milbrandt","sequence":"additional","affiliation":[{"name":"Department of Genetics, Washington University School of Medicine, St. Louis, MO 63110"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark S.","family":"Sands","sequence":"additional","affiliation":[{"name":"Department of Medicine, Washington University School of Medicine, St. Louis, MO 63110;"},{"name":"Department of Genetics, Washington University School of Medicine, St. Louis, MO 63110"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"341","published-online":{"date-parts":[[2020,4,6]]},"reference":[{"key":"e_1_3_3_1_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbamcr.2008.12.001"},{"key":"e_1_3_3_2_2","volume-title":"Epidemiology of Lysosomal Storage Diseases: An Overview","author":"Fuller M.","year":"2006","unstructured":"M. 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