{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T00:45:11Z","timestamp":1790469911024,"version":"4.1.0"},"reference-count":70,"publisher":"Wiley","issue":"11","license":[{"start":{"date-parts":[[2016,8,25]],"date-time":"2016-08-25T00:00:00Z","timestamp":1472083200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"NIH","award":["R01 NS055816"],"award-info":[{"award-number":["R01 NS055816"]}]},{"name":"Oscar Rennebohm Foundation"},{"name":"Boespflug Foundation"},{"name":"MEXT KAKENHI","award":["15K09594"],"award-info":[{"award-number":["15K09594"]}]}],"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), or Krabbe disease, is a devastating demyelinating disease that affects both the central and peripheral nervous systems. It is caused by genetic deficiency in the activity of a lysosomal enzyme, galactocerebrosidase (GALC), which is necessary for the maintenance of myelin. Hematopoietic stem cell transplantation (HSCT) including umbilical cord stem cell transplantation is the only effective therapy available to date. HSCT significantly prolongs the life span of patients with GLD when performed before disease onset, although it is not curative. In HSCT, infiltrating donor\u2010derived macrophages are thought to indirectly supply the enzyme (called \u201ccross\u2010correction\u201d) to the host's myelinating cells. Given the limitation in treating GLD, it is hypothesized that remyelinating demyelinated axons with GALC\u2010competent myelinating cells by transplantation will result in more stable myelination than endogenous myelin repair supported by GALC cross\u2010correction. Transplantation of myelin\u2010forming cells in a variety of animal models of dysmyelinating and demyelinating disorders suggests that this approach is promising in restoring saltatory conduction and protecting neurons by providing new healthy myelin. However, GLD is one of the most challenging diseases in terms of the aggressiveness of the disease and widespread pathology. Experimental transplantation of myelin\u2010forming cells in the brain of a mouse model of GLD has been only modestly effective to date. Thus, a practical strategy for myelin repair in GLD would be to combine the rapid and widespread cross\u2010correction of GALC by HSCT with the robust, stable myelination provided by transplanted GALC\u2010producing myelin\u2010forming cells. This short review will discuss such possibilities. \u00a9 2016 Wiley Periodicals, Inc.<\/jats:p>","DOI":"10.1002\/jnr.23909","type":"journal-article","created":{"date-parts":[[2016,8,25]],"date-time":"2016-08-25T10:03:07Z","timestamp":1472119387000},"page":"1195-1202","source":"Crossref","is-referenced-by-count":11,"title":["Myelin repair by transplantation of myelin\u2010forming cells in globoid cell leukodystrophy"],"prefix":"10.1002","volume":"94","author":[{"given":"Yoichi","family":"Kondo","sequence":"first","affiliation":[{"name":"Department of Anatomy and Cell Biology Osaka Medical College Takatsuki Osaka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ian D.","family":"Duncan","sequence":"additional","affiliation":[{"name":"Department of Medical Sciences, School of Veterinary Medicine University of Wisconsin\u2010Madison Madison 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