特集 神経疾患治療の進歩2019
運動ニューロン疾患の治療の進歩
2020 年 37 巻 5 号 p. 736-740
詳細
2020 年 37 巻 5 号 p. 736-740
Motor neuron diseases (MND), including amyotrophic lateral sclerosis (ALS), spinal muscular atrophy (SMA), and spinal bulbar muscular atrophy (SBMA) are devastating neurodegenerative diseases that primary affect motor neurons. Nucleic acid–based therapeutics and gene transduction therapies are novel treatment methods for MND, which have the potential to transform these incurable diseases into treatable diseases. This review provides an overview of preclinical and clinical advances in MND research, future issues and prospects, and also summarizes selected key literature on therapeutic approaches in 2019.
