Targeting neurological disorders with antisense oligonucleotides

Neurological disorders represent a major health burden that is increasing in magnitude in parallel with the demographic shift in the age of the population of advanced societies. In the United States alone it is estimated that 3.5 million people are afflicted with Alzheimer’s disease, which typically runs its fatal course over 3-5 years [1]. While advances in medicine have been one of the defining achievements of modern times it is fair to say that these have had little impact on the fortunes of persons afflicted with Alzheimer’s disease, multiple system atrophy, frontotemporal dementia, and the like. Modest success has been achieved with symptomatic treatment; the best example being the treatment of Parkinson’s disease with L-dopa and dopamine agonists.