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Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model
RNA interference (RNAi) gene silencing is a potential therapeutic strategy for dominant retinal degeneration disorders. We used self-complementary (sc) AAV2/8 vector to develop an RNAi-based gene therapy in a dominant retinal degeneration mouse model expressing bovine GCAP1(Y99C). We established an...
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| 主要な著者: | , , , , , , |
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| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
National Academy of Sciences
2011
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3215008/ https://ncbi.nlm.nih.gov/pubmed/22042849 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1112758108 |
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