地中海贫血基因治疗研究进展及思考
Research progress and considerations for thalassemia gene therapy
Note： HSCs—hematopoietic stem cells; Cas9—CRISPR-associated protein 9; sgRNA—single guide RNA; LV—lentiviral; AAV—adeno-associated virus; LNP—lipid nanoparticle.
地中海贫血基因治疗研究进展及思考 |
| 高欣洁, 刘艳, 王大威 |
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Research progress and considerations for thalassemia gene therapy |
| GAO Xinjie, LIU Yan, WANG Dawei |
| 图1 地中海贫血基因治疗策略示意图 Note： HSCs—hematopoietic stem cells; Cas9—CRISPR-associated protein 9; sgRNA—single guide RNA; LV—lentiviral; AAV—adeno-associated virus; LNP—lipid nanoparticle. |
| Fig 1 Schematic diagram of thalassemia gene therapy strategies |
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