2020 Fiscal Year Final Research Report

Development of new nucleic acid medicines for intractable respiratory diseases

Research Project

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Project/Area Number	18H02589
Research Category	Grant-in-Aid for Scientific Research (B)
Allocation Type	Single-year Grants
Section	一般
Review Section	Basic Section 47060:Clinical pharmacy-related
Research Institution	Nagasaki University
Principal Investigator	SASAKI Hitoshi 長崎大学, 病院(医学系), 教授 (00170689)
Co-Investigator(Kenkyū-buntansha)	中村忠博長崎大学, 病院(医学系), 技術職員 (60882199) 川上茂長崎大学, 医歯薬学総合研究科(薬学系), 教授 (20322307) 山下親正東京理科大学, 薬学部薬学科, 教授 (30622188) 黒崎友亮長崎大学, 医歯薬学総合研究科(薬学系), 助教 (00582016) 室高広長崎大学, 病院(医学系), 技術職員 (10832834)
Project Period (FY)	2018-04-01 – 2021-03-31
Keywords	遺伝子デリバリー
Outline of Final Research Achievements	In this study, we developed gene and nucleic acid medicines for lung disease based on lung-targeted nanoparticles by systemic or pulmonary administration. Nanoparticles could be constructed by optimizing the mixing ratio of pDNA, cationic compound, and anionic compound for self-assembly. Nanoparticles showed high gene expression in lung selectively after intravenous and pulmonary administration. In addition, the nanoparticles were also able to adapt to siRNA. As a result of adding nanoparticles containing siRNA to luciferase to luciferase expressing mouse melanoma cells, it was shown that they suppressed the luciferase expression. Furthermore, as a result of intravenous administration of nanoparticles to melanoma lung metastasis model mice, lung metastasis was significantly suppressed as compared with the control.
Free Research Field	製剤学、ドラッグデリバリーシステム
Academic Significance and Societal Importance of the Research Achievements	本研究により、全身投与および経肺投与により選択的に送達できるナノ微粒子の調製に成功した。このナノ微粒子はがん細胞にも良好に取り込まれ、遺伝子発現および抑制をすることができるため、肺がんあるいは肺転移がんに対する遺伝子・核酸医薬開発へ応用することが可能である。また、他の肺疾患にも応用が可能であるため、幅広く医療貢献ができる。