| Project/Area Number |
14370560
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Ophthalmology
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| Research Institution | Kagoshima University |
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Principal Investigator |
SAKAMOTO Taiji Kagoshima University, Graduate School of Medical and Dental Sciences, Professor, 大学院・医歯学総合研究科, 教授 (10235179)
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| Project Period (FY) |
2002 – 2004
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| Project Status |
Completed (Fiscal Year 2004)
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| Budget Amount *help |
¥14,700,000 (Direct Cost: ¥14,700,000)
Fiscal Year 2004: ¥2,600,000 (Direct Cost: ¥2,600,000)
Fiscal Year 2003: ¥4,100,000 (Direct Cost: ¥4,100,000)
Fiscal Year 2002: ¥8,000,000 (Direct Cost: ¥8,000,000)
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| Keywords | gene therapy / SIV vector / inherited disease / ultrasound / monkey / retinal degeneration / triamcinolone / vitrectomy / 網膜 / エレクトロポレーション / マイクロバブル / ウイルスベクター / 網膜色素変性症 / 硝子体 / アデノウイルス / レンチウイルス / 国産ベクター / 染色体治療 |
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| Research Abstract |
Gene therapy is the most innovative and potentially effective therapy for various diseases for which no effective therapy exists, however, there are many problems to be solved before clinical application in Japan. It is because the technology of gene therapy is strictly protected by patents, most of which are owned by non-Japanese companies or individuals. Thinking of socio-economical impact of gene therapy in future medicine, it is necessary for us to develop an original gene therapy technology in Japan. Therefore, we performed the following esperiments.
1)Simian immuno-deficiency virus(SIV)-vector mediated gene therapy for retinal degeneration : SIV-vector which was developed by DYNAVEC (Japan) can transfer genes of interest into a choromosome for a long period. In this study, GFP-gene was injected into the monkey eyes using SIV-vector, resulting in stable expression of GFP for as long as 12 months without morphological and functional damages. No systemic adverse effect was found. Now, the effect of neuro-protective gene transfer such as bFGF and PEDF is under investigation.
2)Ultrasound(US)-mediated gene transfer : US is widely accepted to the current medical fields and its safeness has been established. Thus, we developed an US-mediated gene transfer to eyes. GFP cDNA was injected into the cornea followed by exposure to US and micorbubbles. As a result, 2-dimension-like gene transfer was achieved with no tissue damage. This method is under patent proposal.
3)New surgical technology : Safety maneuver to gene transfer is a key issue to perform human gene therapy. For that, we develop a triamcinolone-assisted vitrectomy, which decreased the incidence of surgical complication s.
All of the above results are original and free from the foreign patent restriction. This will make a great contribution to establish a new ocular gene therapy in Japan.
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