Development of therapeutic sgRNA for multiple myeloma

• Project/Area Number: 24300342
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Partial Multi-year Fund
• Section: 一般
• Research Field: Clinical oncology
• Research Institution: Nigata University of Phermacy and Applied Life Sciences
• Principal Investigator: NASHIMOTO Masayuki 新潟薬科大学, 公私立大学の部局等, 教授 (30228069)
• Co-Investigator(Kenkyū-buntansha): KAWANO Mitsuoki 新潟薬科大学, 応用生命科学部, 助教 (00455338)
• Project Period (FY): 2012-04-01 – 2015-03-31
• Project Status: Completed (Fiscal Year 2014)
• Budget Amount: ¥19,500,000 (Direct Cost: ¥15,000,000、Indirect Cost: ¥4,500,000); Fiscal Year 2014: ¥6,630,000 (Direct Cost: ¥5,100,000、Indirect Cost: ¥1,530,000); Fiscal Year 2013: ¥5,590,000 (Direct Cost: ¥4,300,000、Indirect Cost: ¥1,290,000); Fiscal Year 2012: ¥7,280,000 (Direct Cost: ¥5,600,000、Indirect Cost: ¥1,680,000)
• Keywords: 核酸治療 / 多発性骨髄腫 / アポトーシス / 血液がん / TRUE gene silencing / sgRNA / RNA治療 / gene silencing

Outline of Final Research Achievements

Multiple myeloma is the second most common hematological malignancy, and it is estimated that about 150,000 people in the world would die of myeloma every year. Although median survival is extended by development of new drugs, drug-resistant myeloma cells emerge in most of the patients, resulting in subsequent relapse of cancer. The cause of multiple myeloma is not known, and bone marrow transplantation is the only way to cure, albeit with a low success rate.
Our final destination is finding therapeutic sgRNAs to cure multiple myeloma based on TRUE gene silencing, which we have developed as a gene expression control technology. In this study, we elucidated partly how the candidate therapeutic sgRNA works, and showed that this sgRNA can reduce a tumor growth rate in a mouse model experiment.

Research Products

• [Journal Article] Screening of a heptamer-type sgRNA library for potential therapeutic agents against hematological malignancies. (2014)
  • Author(s): Masayuki Takahashi, Reyad A. Elbarbary, Norihiro Watanabe, Atsushi Goto, Daichi Kamiya, Yoshihiro Watabe, Takayoshi Uchiyama, Miwako Narita, Masuhiro Takahashi, Yoshiaki Takahashi, Noriko Ishihara, Tatsuya Miyazawa, Tetsuo Yoshida, Mitsuoki Kawano, Masato Tamura, Masayuki Nashimoto.
  • Journal Title: Luekemia Research Volume: (印刷中) Issue: 7 Pages: 808-815
  • DOI: 10.1016/j.leukres.2014.03.021
  • Peer Reviewed
• [Presentation] 多発性骨髄腫治療用sgRNA薬の開発 (2014)
  • Author(s): 梨本正之、高橋昌幸、二宮翔、涌井直、石川達矢、石垣泰知、櫻井貴一、中野良輔
  • Organizer: 第１８回バイオ治療法研究会学術集会
  • Place of Presentation: アルファあなぶきホール（香川県県民ホール） 大会議室
  • Year and Date: 2014-12-13
• [Presentation] Development of a Therapeutic sgRNA for Multiple Myeloma Based on TRUE Gene Silencing (2013)
  • Author(s): Masayuki Takahashi, Norihiro Watanabe, Miwako Narita, Masuhiro Takahashi, Mitsuoki Kawano, Masato Tamura, Masayuki Nashimoto
  • Organizer: 14th international myeloma workshop
  • Place of Presentation: 京都国際会議場
• [Remarks] 新潟薬科大学 遺伝子発現制御学研究室
  • URL: http://www.nupals.ac.jp/labo/ap/amage/