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Use of megakaryocytes (MKs)\/platelets for transgene expression may take advantage of their rapid turnover and protective storage in platelets and reduce the risk of activating oncogenes in HSC. Here, we demonstrate that the MK\/platelet lineage is capable of generating\/storing a fully functional lysosomal enzyme and leads to highly efficient, continuous, and versatile transport\/distribution of enzyme into the circulation and major diseased organs. This study provides proof of concept of MK\/platelet as a depot for efficient production, delivery, and effective distribution of lysosomal enzymes.<\/jats:p>","DOI":"10.1073\/pnas.1323155111","type":"journal-article","created":{"date-parts":[[2014,2,4]],"date-time":"2014-02-04T01:33:35Z","timestamp":1391477615000},"page":"2680-2685","update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":16,"title":["Platelets are efficient and protective depots for storage, distribution, and delivery of lysosomal enzyme in mice with Hurler Syndrome"],"prefix":"10.1073","volume":"111","author":[{"given":"Mei","family":"Dai","sequence":"first","affiliation":[{"name":"Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jingfen","family":"Han","sequence":"additional","affiliation":[{"name":"Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Salim S.","family":"El-Amouri","sequence":"additional","affiliation":[{"name":"Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229;"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roscoe O.","family":"Brady","sequence":"additional","affiliation":[{"name":"National Institute of Neurological Disorders and Stroke, National Institutes of Health, Bethesda, MD 20892; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dao","family":"Pan","sequence":"additional","affiliation":[{"name":"Division of Experimental Hematology and Cancer Biology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH 45229;"},{"name":"Department of Pediatrics, University of Cincinnati, Cincinnati, OH 45219"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"341","published-online":{"date-parts":[[2014,2,3]]},"reference":[{"key":"e_1_3_3_1_2","doi-asserted-by":"publisher","DOI":"10.1126\/science.1242551"},{"key":"e_1_3_3_2_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.gene.2013.03.098"},{"key":"e_1_3_3_3_2","doi-asserted-by":"publisher","DOI":"10.1002\/stem.716"},{"key":"e_1_3_3_4_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-09-349993"},{"key":"e_1_3_3_5_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2009.04.001"},{"key":"e_1_3_3_6_2","doi-asserted-by":"publisher","DOI":"10.1172\/JCI26674"},{"key":"e_1_3_3_7_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2009-11-255612"},{"key":"e_1_3_3_8_2","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-02-138214"},{"key":"e_1_3_3_9_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.tins.2011.05.006"},{"key":"e_1_3_3_10_2","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2011.04276.x"},{"key":"e_1_3_3_11_2","doi-asserted-by":"publisher","DOI":"10.1002\/path.3021"},{"key":"e_1_3_3_12_2","volume-title":"The Online Metabolic and Molecular Bases of Inherited Disease","author":"Neufeld ES","year":"2007","unstructured":"ES Neufeld, J Muenzer, The mucopolysaccharidoses. 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