2017 年 34 巻 3 号 p. 258-261
Nucleic acid medicine treatment is a promising technology and improving the efficacy of therapeutic oligonucleotides is still needed for its broad use. Although antisense oligonucleotides (ASOs) and short interfering RNA (siRNA) have been well known as nucleic acid medicine, pharmaceutical developments by using them are not completely free to be done due to patent protection. Therefore, we developed a new technology, third generation oligonucleotide. We named it heteroduplex oligonucleotide (HDO). In addition, our original drug delivery system (DDS) using glucose trasporter–1 (GLUT1) made great enhancement on delivery of oligonucleotides into the brain through blood–brain barrier (BBB). In this symposium, we show above two new technologies we have developed, and outline the nucleic acid medicine treatment for amyotrophic lateral sclerosis (ALS).
