Neurology Central

Alexander disease: could a novel stem cell model provide insight into treatment?

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A scientist from the City of Hope (CA, USA) has developed a stem cell model to assess possible treatments for a rare nervous system disorder known as Alexander disease, which is in the same disease group as Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis.

Alexander disease is a type of leukodystrophy – which is characterized by the destruction of myelin sheath – that primarily affects astrocytes. It is caused by mutations in the astrocytic filament gene GFAP; however, the mechanisms for this remain unclear. In most cases, the mutations are de novo and not inherited from the parents. A small proportion of individuals thought to have Alexander disease do not have a mutation in the GFAP gene, suggesting that there are other cause yet to be identified.

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