黄斑变性
医学
病变
不利影响
脉络膜新生血管
眼科
视网膜色素上皮
病毒载体
眼压
外科
内科学
视网膜
生物
重组DNA
生物化学
基因
作者
Peter A. Campochiaro,Quan Dong Nguyen,Syed Mahmood Shah,Michael L. Klein,Eric R. Holz,Robert N. Frank,David A. Saperstein,Anurag Gupta,J. Timothy Stout,Jennifer Macko,Robert Dibartolomeo,Lisa L. Wei
出处
期刊:Human Gene Therapy
[Mary Ann Liebert]
日期:2006-02-01
卷期号:17 (2): 167-176
被引量:335
标识
DOI:10.1089/hum.2006.17.167
摘要
Twenty-eight patients with advanced neovascular age-related macular degeneration (AMD) were given a single intravitreous injection of an E1-, partial E3-, E4-deleted adenoviral vector expressing human pigment epithelium- derived factor (AdPEDF.11). Doses ranging from 106 to 109.5 particle units (PU) were investigated. There were no serious adverse events related to AdPEDF.11 and no dose-limiting toxicities. Signs of mild, transient intraocular inflammation occurred in 25% of patients, but there was no severe inflammation. Six patients experienced increased intraocular pressure that was easily controlled by topical medication. All adenoviral cultures were negative. At 3 and 6 months after injection, 55 and 50%, respectively, of patients treated with 106–107.5 PU and 94 and 71% of patients treated with 108–109.5 PU had no change or improvement in lesion size from baseline. The median increase in lesion size at 6 and 12 months was 0.5 and 1.0 disk areas in the low-dose group compared with 0 and 0 disk areas in the high-dose group. These data suggest the possibility of antiangiogenic activity that may last for several months after a single intravitreous injection of doses greater than 108 PU of AdPEDF.11. This study provides evidence that adenoviral vector-mediated ocular gene transfer is a viable approach for the treatment of ocular disorders and that further studies investigating the efficacy of AdPEDF.11 in patients with neovascular AMD should be performed.
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