Developmental YAPdeltaC determines adult pathology in a model of spinocerebellar ataxia type 1

Protein prevents slow cell death in brain disorder

22 March 2018

A protein that prevents cell death could reduce the symptoms of a debilitating brain disorder.

Spinocerebellar ataxia (SCA) is an inherited disorder that gradually kills off nerve cells, impairing mobility and often causing abnormal speech and eye movement. There is currently no cure for the disease; treatments just reduce symptoms.

A team including researchers from Tokyo Medical and Dental University studied the effects of YAPdeltaC — a protein known to reduce the gradual death of nerve cells — in mice with SCA. They found that this protein is put out of action by the SCA genetic mutation, Atxn1, in mice during development. Increasing the activity of YAPdeltaC in young mice with the SCA mutation with the chemical doxycycline (Dox) reduced SCA symptoms in adulthood and prolonged their life. In contrast, feeding Dox to adult mice with SCA had no such effects.

Understanding how this protein works during the development stage could lead to treatments for late-onset cell death in SCA and other neurodegenerative diseases.

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References

1. Nature Communications 8, 1864 (2017). doi: 10.1038/s41467-017-01790-z