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Myelin repair by transplantation of myelin-forming cells in globoid cell leukodystrophy

Kondo, Yoichi ; Duncan, Ian D.

Journal of neuroscience research, 2016-11, Vol.94 (11), p.1195-1202 [Periódico revisado por pares]

United States: Blackwell Publishing Ltd

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  • Título:
    Myelin repair by transplantation of myelin-forming cells in globoid cell leukodystrophy
  • Autor: Kondo, Yoichi ; Duncan, Ian D.
  • Assuntos: Animals ; Demyelinating Diseases - etiology ; Demyelinating Diseases - surgery ; Disease Models, Animal ; Galactosylceramidase - deficiency ; Galactosylceramidase - genetics ; Hematopoietic Stem Cell Transplantation - methods ; Humans ; induced pluripotent stem cells ; Krabbe disease ; Leukodystrophy, Globoid Cell - complications ; Leukodystrophy, Globoid Cell - genetics ; Leukodystrophy, Globoid Cell - surgery ; myelin repair ; Myelin Sheath - physiology ; transplantation
  • É parte de: Journal of neuroscience research, 2016-11, Vol.94 (11), p.1195-1202
  • Notas: ark:/67375/WNG-V7NJ3S57-4
    Boespflug Foundation
    NIH - No. R01 NS055816
    MEXT KAKENHI - No. 15K09594
    istex:DAAB17AF340E2FE0DB7B69215D6049B4BC658041
    ArticleID:JNR23909
    Oscar Rennebohm Foundation
    Support/grant information: The works noted from our labs were supported by NIH grant (R01 NS055816), the Oscar Rennebohm Foundation, the Boespflug Foundation, and MEXT KAKENHI (15K09594).
    SIGNIFICANCE Globoid cell leukodystrophy (GLD), or Krabbe disease, is a genetic demyelinating disease in which most affected children succumb to systemic, progressive neurological disabilities by 2 years of age. Hematopoietic stem cell transplantation, which indirectly provides the host's myelinating cells with the missing enzyme, galactocerebrosidase, has serious limitations. This Mini‐Review proposes potential strategies for treating GLD, specifically focusing on transplantation of the progenitors of myelin‐forming cells to repair lost myelin. We discuss the pros and cons of such transplantation and suggest that combining other approaches will turn out to be realistic and most effective.
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  • Descrição: 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‐derived macrophages are thought to indirectly supply the enzyme (called “cross‐correction”) to the host's myelinating cells. Given the limitation in treating GLD, it is hypothesized that remyelinating demyelinated axons with GALC‐competent myelinating cells by transplantation will result in more stable myelination than endogenous myelin repair supported by GALC cross‐correction. Transplantation of myelin‐forming 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‐forming 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‐correction of GALC by HSCT with the robust, stable myelination provided by transplanted GALC‐producing myelin‐forming cells. This short review will discuss such possibilities. © 2016 Wiley Periodicals, Inc.
  • Editor: United States: Blackwell Publishing Ltd
  • Idioma: Inglês
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