Subretinal Gene Therapy for Treatment of Retinal and Choroidal Vascular Diseases

IF 4.2 1区 医学 Q1 OPHTHALMOLOGY American Journal of Ophthalmology Pub Date : 2025-09-01 Epub Date: 2024-12-07 DOI:10.1016/j.ajo.2024.12.002
Arshad M. Khanani , Aamir A. Aziz , Zoha A. Khanani , Hannah Khan , Ohidul Mojumder , Humza Sulahria , Ibrahim Khanani , Huma Khan , Greggory M. Gahn , Kapil Mishra
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治疗视网膜和脉络膜血管疾病的视网膜下基因疗法。
目的:本文综述了视网膜下基因治疗视网膜血管疾病的研究进展,包括表达可溶性血管内皮生长因子(VEGF)受体1的腺相关病毒(AAV) 2载体AVA-101、表达抗VEGF- a抗体片段的AAV8载体ABBV-RGX-314和表达阻断VEGF家族成员和血管生成素2的重组蛋白的AAV8载体EXG102-031。结论:视网膜下注射是一种常用的基因治疗药物的递送途径,理论上可以提供相对免疫特权,从而降低炎症风险,同时在光感受器和视网膜色素上皮中提供高转基因表达。在I期和IIa期试验中,视网膜下注射AVA-101显示出安全性和耐受性,但未能维持视力和控制渗出。相比之下,一些剂量的ABBV-RGX-314的视网膜下注射显示出控制渗出和维持视力的证据,以及安全性和耐受性,导致两项正在进行的关键性试验比较了两种不同剂量的ABBV-RGX-314的视网膜下递送与玻璃体内注射0.5mg雷尼单抗或2mg阿非利塞普。这些初步结果是一个令人鼓舞和欢迎的发展,在寻找有效的,长期治疗视网膜血管疾病。
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来源期刊
CiteScore
9.20
自引率
7.10%
发文量
406
审稿时长
36 days
期刊介绍: The American Journal of Ophthalmology is a peer-reviewed, scientific publication that welcomes the submission of original, previously unpublished manuscripts directed to ophthalmologists and visual science specialists describing clinical investigations, clinical observations, and clinically relevant laboratory investigations. Published monthly since 1884, the full text of the American Journal of Ophthalmology and supplementary material are also presented online at www.AJO.com and on ScienceDirect. The American Journal of Ophthalmology publishes Full-Length Articles, Perspectives, Editorials, Correspondences, Books Reports and Announcements. Brief Reports and Case Reports are no longer published. We recommend submitting Brief Reports and Case Reports to our companion publication, the American Journal of Ophthalmology Case Reports. Manuscripts are accepted with the understanding that they have not been and will not be published elsewhere substantially in any format, and that there are no ethical problems with the content or data collection. Authors may be requested to produce the data upon which the manuscript is based and to answer expeditiously any questions about the manuscript or its authors.
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