Fabry disease is caused by a deficiency of lysosomal alpha-galactosidase A (AGA).
Stable transfection of shRNA silenced AGA expression in the neuronal cell line LA-N-2.
Gene-silenced LA-N-2 cells have reduced AGA activity and accumulate Gb3.
Gene-silenced LA-N-2 show impaired neurotransmitter release and poor growth.