GeneticsbioRxivPreprint
In-utero rescue of neurological dysfunction in a mouse model of Wiedemann-Steiner syndrome
Tinna Reynisdottir, Kimberley Jade Anderson, Andrew Brinn…·Hans Tomas Bjornsson
This study demonstrates that in-utero restoration of KMT2A in a mouse model of WDSTS rescues neurological phenotypes, suggesting a therapeutic window during mid-gestation.
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