{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T01:00:56Z","timestamp":1777510856111,"version":"3.51.4"},"reference-count":26,"publisher":"SAGE Publications","issue":"5","license":[{"start":{"date-parts":[[2000,9,1]],"date-time":"2000-09-01T00:00:00Z","timestamp":967766400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Cell Transplant"],"published-print":{"date-parts":[[2000,9]]},"abstract":"<jats:p>Cell therapy with human amniotic epithelial (HAE) cells was developed as an alternative method for enzyme replacement therapy in congenital lysosomal storage disorders, but only limited therapeutic efficacy has been reported. A major drawback is insufficient production and secretion of lysosomal enzymes from HAE cells. In this study, we infected HAE cells with an E1-deleted adenoviral vector expressing human \u03b2-glucuronidase (GUSB), and generated cells overexpressing GUSB by a hundred times as much as endogenous GUSB in untreated HAE cells. GUSB secreted from the gene-transferred HAE cells were efficiently transported to murine fibroblasts with endocytosis mediated by mannose-6-phosphate receptors. The cells were administered into the spleen of the mice with the lysosomal storage disease mucopolysaccharidosis type VII (B6\/MPSVII). Approximately 10\u201315% of the normal GUSB activity was detected in both liver and spleen 7 days after the cell administration. Histopathological examination showed that lysosomal enlargement in tissue macrophages in the liver and the spleen had disappeared by day 14. These results suggest that transplantation of the HAE cells transduced with adenoviral vectors can be employed for the treatment of congenital lysosomal storage disorders.<\/jats:p>","DOI":"10.1177\/096368970000900515","type":"journal-article","created":{"date-parts":[[2017,6,22]],"date-time":"2017-06-22T15:14:03Z","timestamp":1498144443000},"page":"687-692","source":"Crossref","is-referenced-by-count":19,"title":["Phenotype Correction in Murine Mucopolysaccharidosis Type VII by Transplantation of Human Amniotic Epithelial Cells after Adenovirus-Mediated Gene Transfer"],"prefix":"10.1177","volume":"9","author":[{"given":"Motomichi","family":"Kosuga","sequence":"first","affiliation":[{"name":"Departments of Genetics National Children's Medical Research Center, Tokyo, Japan"},{"name":"Department of Pediatrics, Keio University School of Medicine, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Satori","family":"Takahashi","sequence":"additional","affiliation":[{"name":"Departments of Genetics National Children's Medical Research Center, Tokyo, Japan"},{"name":"Department of Science and Technology, Science University of Tokyo, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kyoko","family":"Sasaki","sequence":"additional","affiliation":[{"name":"Departments of Genetics National Children's Medical Research Center, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shin","family":"Enosawa","sequence":"additional","affiliation":[{"name":"Experimental Surgery, National Children's Medical Research Center, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Xiao-Kang","family":"Li","sequence":"additional","affiliation":[{"name":"Experimental Surgery, National Children's Medical Research Center, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sanae","family":"Okuyama","sequence":"additional","affiliation":[{"name":"Experimental Surgery, National Children's Medical Research Center, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Masayuki","family":"Fujino","sequence":"additional","affiliation":[{"name":"Experimental Surgery, National Children's Medical Research Center, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Seiichi","family":"Suzuki","sequence":"additional","affiliation":[{"name":"Experimental Surgery, National Children's Medical Research Center, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Masao","family":"Yamada","sequence":"additional","affiliation":[{"name":"Departments of Genetics National Children's Medical Research Center, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nobutake","family":"Matsuo","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Keio University School of Medicine, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Norio","family":"Sakuragawa","sequence":"additional","affiliation":[{"name":"Department of Inherited and Metabolic Diseases, National Institute of Neuroscience, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Torayuki","family":"Okuyama","sequence":"additional","affiliation":[{"name":"Departments of Genetics National Children's Medical Research Center, Tokyo, Japan"},{"name":"Department of Pediatrics, Keio University School of Medicine, Tokyo, Japan"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","published-online":{"date-parts":[[2017,6,22]]},"reference":[{"key":"bibr1-096368970000900515","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(81)91212-5"},{"key":"bibr2-096368970000900515","doi-asserted-by":"publisher","DOI":"10.1097\/00062752-199704010-00004"},{"key":"bibr3-096368970000900515","doi-asserted-by":"publisher","DOI":"10.1002\/ajmg.1320440430"},{"key":"bibr4-096368970000900515","doi-asserted-by":"publisher","DOI":"10.1089\/10430349950019219"},{"key":"bibr5-096368970000900515","doi-asserted-by":"publisher","DOI":"10.1016\/0167-4889(83)90068-X"},{"key":"bibr6-096368970000900515","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V94.6.2142"},{"key":"bibr7-096368970000900515","doi-asserted-by":"crossref","first-page":"22627","DOI":"10.1016\/S0021-9258(18)41574-8","volume":"268","author":"Islam M. 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