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RPGR gene therapy presents challenges in cloning the coding sequence
INTRODUCTION: Currently, there are three Phase I/II clinical trials based on gene therapy ongoing to test different AAV.RPGR or deleted RPGR vectors on patients affected by X-linked retinitis pigmentosa. These three vectors differ in the adeno-associated viral (AAV) vector capsid used, and the codin...
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| 出版年: | Expert Opin Biol Ther |
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| 主要な著者: | , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
2020
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| 主題: | |
| オンライン･アクセス: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7104355/ https://ncbi.nlm.nih.gov/pubmed/31612744 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1080/14712598.2020.1680635 |
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