Stem cells as a model for Huntington's disease?

Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder caused by an expanded polyglutamine repeat in the huntingtin gene. The neuropathology of HD is characterized by the decline of a specific neuronal population within the brain, the striatal medium spiny neurons (MSNs). The origins of this extreme vulnerability remain unknown.

It is possible to produce striatal neurons from stem cells, e.g. from induced pluripotent stem cells (hiPS cells). But previous protocols to obstain these cells show big heterogeneity in the obtained neurons.

We have studied the role of sodium channels in Huntington's disease and have been able to show the importance of adapting and optimising differentiation protocols in order to achieve reliable results.

The results of this study can be found in a Scientific Reports paper published in 2021:

⇒ The difficulty to model Huntington's disease in vitro using striatal medium spiny neurons differentiated from human induced pluripotent stem cells

 

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