Several inherited metabolic diseases with hepatocerebral phenotype might benefit from a similar dual targeting approach such as mitochondrial diseases caused by nuclear genetic defects (e.g. POLG1, MPV17, DGUOK genes) and some lysosomal storage disorders (e.g. neuronopathic Gaucher disease, mucopolysaccharidosis type I and II). Here, MPV17 is linked to lysosomal storage disease.