We tested whether sodium phenylbutyrate (4PBA) could rescue the function of mutant bestrophin 1 associated with autosomal dominant and recessive disease.
Here we describe how selection for specific mutations that increase HSC competitive fitness, in conjunction with additional endogenous and environmental changes, drives leukaemic transformation.
Major breakthroughs in stem cell differentiation to therapeutically relevant cell types have led us to the doorstep of novel cell therapies that can replace disease management with a cure.
