著者
西田 陽一郎 横田 隆徳
出版者
日本神経治療学会
雑誌
神経治療学 (ISSN:09168443)
巻号頁・発行日
vol.34, no.3, pp.258-261, 2017 (Released:2017-10-14)
参考文献数
9

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).

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mRNA医薬でALSの治療案もあるね。 https://t.co/tOLa0zQIow https://t.co/NKMY2KOUvN
レナセラピューティックス (HDO) 横田教授により見出されたHDO(ヘテロ2本鎖核酸)は、従来のアンチセンス・siRNAよりも薬効面・動態面で優れる。脳へのデリバリー技術も有しており(東大片岡教授の技術) ALS等の中枢疾患への応用も期待される。https://t.co/esePHTEa43

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