However, with the increasing knowledge of antisense oligonucleotide (ASO)-mediated therapies for the treatment of rare neurological disorders, it is possible to envisage the combination of specific ASOs, which can reduce the sole expression of the mRNAs carrying spastin missense mutations, with spastin-elevating approaches to increase the post-translational stability of the wild-type variant. The gene discussed is SPAST; the disease is nervous system disorder.