Treatment of neurological diseases by innovative novel nucleic acid medicine

Fumiko Furukawa, Tetsuya Nagata, Nobuo Sanjo, Takanori Yokota

Department of Neurology and Neurological Science, Tokyo Medical and Dental University

Alzheimer's disease (AD) is the most common form of dementia, affecting up to 50% of all patients with dementia. However, the current standard of care for AD is limited to symptomatic therapies which provide only temporary improvement in cognitive and behavioral symptoms. The development of disease-modifying therapies (DMTs) for AD are highly anticipated. Recently, antisense oligonucleotides (ASOs) therapy─with their diverse functionality and high target specificity─has emerged as an exciting and promising strategy for the neurological diseases and the clinical trials for spinal muscular atrophy and Huntington's disease are ongoing. Here we review the disease-related genes of AD as potential target of ASOs and development of ASOs for the treatment of dementia.

Address correspondence to Dr. Fumiko Furukawa, Department of Neurology and Neurological Science, Tokyo Medical and Dental University (Bunkyo-ku, Tokyo 113-8519, Japan)