Radically truncated MeCP2 rescues Rett syndrome-like neurological defects

Tillotson, R., Selfridge, J., Koerner, M.V., Gadalla, K.K.E., Guy, J., De Sousa, D., Hector, R.D., Cobb, S.R., Bird, A.

Nature (2017) 550, 398–401

Rett syndrome is a profound neurological disorder caused by mutations affecting the MeCP2 protein (vertical strokes). We find that its essential functions can be performed by two short domains and gene therapy using this mini-MeCP2 rescues many Rett-like defects in mice.  

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