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Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus
Usher 1 patients are born profoundly deaf and then develop retinal degeneration. Thus they are readily identified prior to the onset of retinal degeneration, making gene therapy a viable strategy to prevent their blindness. Here, we have investigated the use of adeno-associated viruses (AAV) for the...
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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: |
2013
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3640772/ https://ncbi.nlm.nih.gov/pubmed/23344065 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/gt.2013.3 |
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