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Stem cells in genetic myelin disorders.

Authors
  • Kemp, Kevin
  • Mallam, Elizabeth
  • Scolding, Neil
  • Wilkins, Alastair
Type
Published Article
Journal
Regenerative medicine
Publication Date
May 01, 2010
Volume
5
Issue
3
Pages
425–439
Identifiers
DOI: 10.2217/rme.10.10
PMID: 20455653
Source
Medline
License
Unknown

Abstract

The genetic myelin disorders are a range of diseases that manifest with severe neurological problems, often from infancy. It has been postulated for some time that stem cells might be an effective treatment for these disorders, primarily as agents to restore dysfunctional or lost myelin. Stem cells, however, may offer a wider range of therapeutic potential, for instance as vehicles to replace abnormal enzymes or genes, or to provide trophic support for residual CNS tissue. This article will review several of the more common genetic myelin disorders and currently available therapies, including bone marrow transplantation for adrenoleukodystrophy. Specific stem cell subtypes and their relevance to potential therapeutic use will be discussed and stem cell transplantation in animal model studies will also be reviewed.

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