Therapeutic approach for ocular amyloidosis by ocular-targeted multi-functional drug delivery system
| Project/Area Number |
17H06970
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| Research Category |
Grant-in-Aid for Research Activity Start-up
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| Allocation Type | Single-year Grants |
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| Research Field |
Medical pharmacy
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| Research Institution | Kumamoto University |
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Principal Investigator |
Hayashi Yuya 熊本大学, 医学部附属病院, 特別研究員 (50806429)
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| Project Period (FY) |
2017-08-25 – 2019-03-31
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| Project Status |
Completed (Fiscal Year 2018)
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| Budget Amount *help |
¥2,730,000 (Direct Cost: ¥2,100,000、Indirect Cost: ¥630,000)
Fiscal Year 2018: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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| Keywords | 眼アミロイドーシス / ドラッグデリバリーシステム / 核酸医薬 / シクロデキストリン / トランスサイレチン / デンドリマー / DDS / siRNA / アミロイドーシス |
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| Outline of Final Research Achievements |
In the present study, we developed ocular-targeted multi-functional siRNA carrier for the therapeutic use of transthyretin (TTR)-related ocular amyloidosis, and evaluated the inhibitory effect of TTR expression and amyloid fibril formation.
Ocular-targeted multi-functional carrier/TTR-siRNA complex elicited TTR silencing effects in vitro and in vivo. Furthermore, ocular-targeted multi-functional siRNA carrier showed potent inhibition of TTR amyloid fibril formation. In conclusion, the present study demonstrated the potentials of ocular-targeted multi-functional siRNA carrier as a novel TTR-related ocular amyloidosis therapy.
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| Academic Significance and Societal Importance of the Research Achievements |
これまでにTTR-FAPにおける全身症状に対しては肝臓移植、内服薬、核酸医薬製剤が開発されたものの、眼アミロイドーシス症状に対する治療法は皆無であり、失明に陥ってしまう多くの患者がいる現状は何としてでも解決しなければならない。本研究で得られた眼特異的多機能型DDSにより眼におけるTTR産生およびアミロイド線維形成を抑制可能という成果は、眼アミロイドーシスの新たな治療法につながる重要な知見であると確信する。
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Report
(3 results)
Research Products
(4 results)
