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视网膜退行性疾病基因治疗的临床试验概况:基于Trialtrove数据库的分析

Clinical trial landscape of gene therapy for retinal degenerative diseases: an analysis based on the Trialtrove database.

作者信息

Cheng Shizhou, Xing Chuyun, Chen Zijian, Zhang Shu, Chen Wanghao

机构信息

Department of Ophthalmology, The First Affiliated Hospital of Yangtze University, Jingzhou, 434000, Hubei Province, China.

Department of Pain Management, The First Affiliated Hospital of Yangtze University, Jingzhou, 434000, Hubei Province, China.

出版信息

Stem Cell Res Ther. 2025 May 28;16(1):257. doi: 10.1186/s13287-025-04387-2.

DOI:10.1186/s13287-025-04387-2
PMID:40437529
Abstract

Gene therapy has changed the way retinal degenerative diseases are explored and treated. Analyzing past and ongoing clinical trials using gene therapy for retinal degenerative diseases will provide important insight into academic, medical, and other communities. This study retrospectively analyzed 209 clinical trials in the Trialtrove database initiated before 2025.

摘要

基因治疗改变了视网膜退行性疾病的研究和治疗方式。分析过去和正在进行的使用基因治疗视网膜退行性疾病的临床试验,将为学术界、医学界和其他团体提供重要的见解。本研究回顾性分析了Trialtrove数据库中在2025年前启动的209项临床试验。

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本文引用的文献

1
The global status of bioequivalence trials: a comprehensive clinical trial landscape analysis based on the Trialtrove database.生物等效性试验的全球现状:基于Trialtrove数据库的全面临床试验格局分析
Drug Discov Today. 2024 Dec;29(12):104223. doi: 10.1016/j.drudis.2024.104223. Epub 2024 Nov 7.
2
Gene Therapies in Clinical Development to Treat Retinal Disorders.处于临床开发阶段的用于治疗视网膜疾病的基因疗法。
Mol Diagn Ther. 2024 Sep;28(5):575-591. doi: 10.1007/s40291-024-00722-0. Epub 2024 Jul 2.
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Outcome differences by sex in oncology clinical trials.
肿瘤学临床试验中性别导致的结果差异。
Nat Commun. 2024 Mar 23;15(1):2608. doi: 10.1038/s41467-024-46945-x.
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Gene augmentation therapy to rescue degenerative photoreceptors in a Cwc27 mutant mouse model.基因增强疗法挽救 Cwc27 突变小鼠模型中变性的光感受器
Exp Eye Res. 2023 Sep;234:109596. doi: 10.1016/j.exer.2023.109596. Epub 2023 Jul 20.
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Clinical development of mRNA therapies against solid tumors.mRNA 疗法在实体瘤治疗中的临床进展。
J Hematol Oncol. 2023 Jul 18;16(1):75. doi: 10.1186/s13045-023-01457-x.
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Gene Therapy for Retinal Degenerative Diseases: Progress, Challenges, and Future Directions.基因治疗视网膜退行性疾病:进展、挑战与未来方向。
Invest Ophthalmol Vis Sci. 2023 Jun 1;64(7):39. doi: 10.1167/iovs.64.7.39.
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Current challenges in biotherapeutic particles manufacturing.生物治疗颗粒制造的当前挑战。
Expert Opin Biol Ther. 2020 May;20(5):451-465. doi: 10.1080/14712598.2020.1693541. Epub 2019 Nov 27.
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Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here?基于 Luxturna(以及 Zolgensma 和 Glybera)的腺相关病毒基因治疗的现状:我们在哪里,我们是如何走到这一步的?
Annu Rev Virol. 2019 Sep 29;6(1):601-621. doi: 10.1146/annurev-virology-092818-015530. Epub 2019 Jul 5.
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Seeing the Light after 25 Years of Retinal Gene Therapy.二十五载视网膜基因治疗终见光明。
Trends Mol Med. 2018 Aug;24(8):669-681. doi: 10.1016/j.molmed.2018.06.006. Epub 2018 Jul 5.