{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,20]],"date-time":"2026-09-20T12:57:07Z","timestamp":1789909027392,"version":"4.0.1"},"reference-count":52,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2016,9,17]],"date-time":"2016-09-17T00:00:00Z","timestamp":1474070400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100000054","name":"National Cancer Institute","doi-asserted-by":"crossref","award":["P30 CA023074"],"award-info":[{"award-number":["P30 CA023074"]}],"id":[{"id":"10.13039\/100000054","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Neuroscience Research"],"published-print":{"date-parts":[[2016,11]]},"abstract":"<jats:p>Globoid cell leukodystrophy (GLD) is a progressive neurodegenerative disorder caused by the deficiency of galactocerebrosidase (GALC), resulting in accumulation of toxic metabolites in neural tissues. Clinically variable based on age of onset, infantile GLD is generally a rapidly fatal syndrome of progressive neurologic and cognitive decline, whereas later\u2010onset GLD has a more indolent, protracted clinical course. Animal models, particularly the twitcher mouse, have allowed investigation of both the pathophysiology of and the potential treatment modalities for GLD. Cellular therapy for GLD, notably hematopoietic cell transplantation (HCT; transplantation of bone marrow, peripheral blood stem cells, or umbilical cord blood cells) from a normal related or unrelated allogeneic donor provides a self\u2010renewing source of GALC in donor\u2010derived cells. The only currently available treatment option in human GLD, allogeneic HCT, can slow the progression of the disease and improve survival, especially when performed in presymptomatic infants. Because persistent neurologic dysfunction still occurs after HCT in GLD, preclinical studies are evaluating combinations of HCT with other treatment modalities. \u00a9 2016 Wiley Periodicals, Inc.<\/jats:p>","DOI":"10.1002\/jnr.23782","type":"journal-article","created":{"date-parts":[[2016,9,17]],"date-time":"2016-09-17T11:22:31Z","timestamp":1474111351000},"page":"1180-1188","source":"Crossref","is-referenced-by-count":9,"title":["Cellular transplant therapies for globoid cell leukodystrophy: Preclinical and clinical observations"],"prefix":"10.1002","volume":"94","author":[{"given":"Keri R.","family":"Maher","sequence":"first","affiliation":[{"name":"University of Arizona Cancer Center Tucson Arizona"},{"name":"Department of Medicine University of Arizona College of Medicine Tucson Arizona"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew M.","family":"Yeager","sequence":"additional","affiliation":[{"name":"University of Arizona Cancer Center Tucson Arizona"},{"name":"Department of Medicine University of Arizona College of Medicine Tucson Arizona"},{"name":"Department of Pediatrics University of Arizona College of Medicine Tucson Arizona"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2016,9,17]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/sj.bmt.1700665"},{"key":"e_1_2_11_3_1","first-page":"238","article-title":"An update on in utero bone marrow transplantation for Krabbe disease","volume":"13","author":"Blakemore KJ","year":"1999","journal-title":"J Mol Neurosci"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1034\/j.1399-3046.2002.02026.x"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2005.05585.x"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/103.3.695"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1097\/GIM.0b013e3181a16e04"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa042604"},{"key":"e_1_2_11_9_1","doi-asserted-by":"crossref","first-page":"932","DOI":"10.1001\/jama.1997.03550110070039","article-title":"In utero hematopoietic stem cell transplantation","volume":"278","author":"Flake AW","year":"1997","journal-title":"A status report. 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