There are multiple preclinical studies reporting the therapeutic effects of ASOs with new target mRNAs in neurological conditions, including amyloid-β precursor protein in Alzheimer’s disease and Down syndrome; PMP22 in Charcot–Marie–Tooth type 1A; DUX4 overexpression in FSHD; expanded trinucleotide repeats in fragile X-associated tremor/ataxia syndrome and Huntington’s disease; HuR levels in neuropathic pain; and inhibition of inflammation in spinal cord injury and traumatic brain injury (Table 4) [74,75,87,135,271,272,273,274,275,276,277,278,279,280,281]. The gene discussed is DUX4; the disease is early-onset autosomal dominant Alzheimer disease.