2013 Fiscal Year Final Research Report
gene therapy using siena technology
Project/Area Number |
23792014
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Research Category |
Grant-in-Aid for Young Scientists (B)
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Allocation Type | Multi-year Fund |
Research Field |
Ophthalmology
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Research Institution | Nippon Medical School |
Principal Investigator |
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Project Period (FY) |
2011 – 2013
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Keywords | 網膜 / 遺伝子治療 / AAVベクター / siRNA / VEGF / 脈絡膜新生血管 |
Research Abstract |
To assess the feasibility of a gene therapeutic approach to treating choroidal neovascularization (CNV), we generated an adeno-associated virus type 8 vector (AAV8) encoding a small interfering RNA (siRNA) targeting vascular endothelial growth factor (VEGF), and determined its ability to inhibit angiogenesis. We made AAV8/SmVEGF-2. We injected AAV8/SmVEGF-2 or control vector (AAV8/GFP) into the subretinal space in C57BL/6 mice. The eyes were removed for flat mount analysis of CNV surface area. Subretinal delivery of AAV8/SmVEGF-2 significantly diminished CNV at the laser lesions, as compared to AAV8/GFP Using ELISA, we found that VEGF levels were reduced by approximately half in AAV8/SmVEGF-2 treated eyes. These results suggest that siRNA-VEGF can be expressed across the retina and that long-term suppression of CNV is possible through the use of stable AAV-mediated siRNA-VEGF expression.
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Research Products
(29 results)
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[Journal Article] Rapid and Transient Upregulation of CCL11 (Eotaxin-1) in Mouse Ovary During Terminal Stages of Follicular Development2012
Author(s)
Kuwabara Y, Katayama A, Igarashi T, Tomiyama R, Piao H, Kaneko R, Abe T, Mine K, Akira S, Orimo H, Takeshita T
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Journal Title
Am J Reprod Immunol
Volume: 67(5)
Pages: 358-368
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[Journal Article] A new mutation in RP1L1 gene in occult macular dystrophy patient associated with a depolarizing-pattern of focal macular ERG2012
Author(s)
Kabuto T, Takahashi H, Goto-Fukuura Y, Igarashi T, Akahori M, Kameya S, Iwata T, Mizota A, Yamaki K, Miyake Y, Takahashi H
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Journal Title
Molecular Vision
Volume: 18
Pages: 1031-1039
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