2013 Fiscal Year Final Research Report
Development of novel gene therapy targeting age-related macular degeneration
| Project/Area Number |
23659813
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Ophthalmology
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| Research Institution | Nagasaki University |
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Principal Investigator |
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| Project Period (FY) |
2011 – 2013
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| Keywords | 加齢黄斑変性症 / 遺伝子治療 |
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| Research Abstract |
In this study, we developed the safe and efficient novel ocular gene delivery system to the age-related macular degeneration. We successfully prepared the stable nanoparticles (pDNA-PEI complex: PEI complex, pDNA-PEI-gamma-PGA complex: PGA complex, pDNA-PEI-CS complex: CS complex) by optimization of the mix ratio. The PGA complex and CS complex showed high gene expression comparable to PEI complex. While PEI complex showed severe agglutination of vitreous body and cytotoxicity, PGA complex and CS complex showed no agglutination of vitreous body and no cytotoxicity. We also developed siRNA-PEI-gamma-PGA (siPGA complexes). The siPGA complexes showed high silencing effect comparable to commercial vector (lipofectamine) without cytotoxicity.
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Research Products
(16 results)
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[Journal Article] Ternary complex of plasmid DNA with protamine and γ-polyglutamic acid for biocompatible gene delivery system2013
Author(s)
Kanda K, Kodama Y, Kurosaki T, Imamura M, Nakagawa H, Muro T, Higuchi N, Nakamura T, Kitahara T, Honda M, Sasaki H
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Journal Title
Biol Pharm Bull
Volume: 36(11)
Pages: 1794-9
Peer Reviewed
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[Journal Article] Ocular gene delivery systems using ternary complexes of plasmid DNA, polyethylenimine, and anionic polymers2013
Author(s)
Kurosaki T, Uematsu M, Shimoda K, Suzuma K, Nakai M, Nakamura T, Kitahara T, Kitaoka T, Sasaki H
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Journal Title
Biol Pharm Bull
Volume: 36(1)
Pages: 96-101
Peer Reviewed
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