期刊
REGENERATIVE MEDICINE
卷 5, 期 3, 页码 425-439出版社
FUTURE MEDICINE LTD
DOI: 10.2217/RME.10.10
关键词
hematopoietic; induced pluripotent stem; leukodystrophy mesenchymal; myelin; oligodendrocyte; remyelination; stem cell
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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