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本文引用的文献

1
The menace of severe adverse events and deaths associated with viral gene therapy and its potential solution.病毒基因治疗相关的严重不良事件和死亡的威胁及其潜在解决方案。
Med Res Rev. 2024 Sep;44(5):2112-2193. doi: 10.1002/med.22036. Epub 2024 Mar 28.
2
Adeno-associated virus vector as a platform for gene therapy delivery.腺相关病毒载体作为基因治疗传递的平台。
Nat Rev Drug Discov. 2019 May;18(5):358-378. doi: 10.1038/s41573-019-0012-9.
3
Gene therapy comes of age.基因治疗走向成熟。
Science. 2018 Jan 12;359(6372). doi: 10.1126/science.aan4672.
4
Gendicine: the first commercial gene therapy product.今又生：首个商业化基因治疗产品。
Hum Gene Ther. 2005 Sep;16(9):1014-5. doi: 10.1089/hum.2005.16.1014.
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Stage I clinical trial of gene therapy for hemophilia B.
Sci China B. 1993 Nov;36(11):1342-51.
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Gene therapy for human genetic disease?用于人类遗传疾病的基因治疗？
Science. 1972 Mar 3;175(4025):949-55. doi: 10.1126/science.175.4025.949.

Gene therapy in China: From a clinician's perspective.

作者信息

Wang Xiaole, Peng Jing

机构信息

Department of Pediatrics, Xiangya Hospital of Central South University, Changsha, Hunan Province, China.

Department of Pediatrics, Xiangya Hospital of Central South University, Changsha, Hunan Province, China; Clinical Research Center for Children Neurodevelopmental Disabilities of Hunan Province, Changsha, China.

出版信息

Mol Ther. 2025 Jun 4;33(6):2330-2331. doi: 10.1016/j.ymthe.2025.01.018. Epub 2025 Jan 24.

DOI:10.1016/j.ymthe.2025.01.018
PMID:39862862
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12172180/
Abstract
摘要