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学科主题: 临床医学
题名:
Prevalence of Neutralizing Factors Against Adeno-Associated Virus Types 2 in Age-Related Macular Degeneration and Polypoidal Choroidal Vasculopathy
作者: Cheng, Yong1; Huang, Lvzhen1; Li, Xiaoxin1; Qi, Huijun1; Zhou, Peng1; Yu, Wenzhen1; Jiang, Yide A.2; Wadsworth, Samuel2; Scaria, Abraham2
关键词: Neutralizing factors ; adeno-associated virus types 2 ; age related macular degeneration ; polypoidal choroidal vasculopathy
刊名: CURRENT GENE THERAPY
发表日期: 2013-06-01
卷: 13, 期:3, 页:182-188
收录类别: SCI
文章类型: Article
WOS标题词: Science & Technology
类目[WOS]: Genetics & Heredity
研究领域[WOS]: Genetics & Heredity
关键词[WOS]: MEDIATED GENE-TRANSFER ; ENDOTHELIAL GROWTH-FACTOR ; RETINAL-PIGMENT EPITHELIUM ; INTRAVITREAL BEVACIZUMAB ; PHOTODYNAMIC THERAPY ; VISUAL IMPAIRMENT ; JAPANESE PATIENTS ; IMMUNE-RESPONSES ; VIRAL VECTORS ; ANTIBODIES
英文摘要:

Adeno-associated virus type 2 (AAV2) mediated gene therapy providing a potential treatment in the eye. However, immune responses can limit virally mediated gene transfer and therapy. To assess preexisting AAV2 neutralizing factors (NF) titers in peripheral blood and the vitreous in patients with age-related macular degeneration (AMD) and polypoidal choroidal vasculopathy (PCV). 130 subjects were enrolled: 50 with neovascular AMD, 30 with PCV, and 50 controls. The serum and the vitreous were obtained for AAV2 NF assay. We found AAV2 NF are present in all of AMD, PCV patients and controls we tested. There were no significant differences in prevalence of NAb in serum between AMD, PCV and controls (P=0.999). There was no correlation between NF in serum and in vitreous (P>0.05), and NF in vitreous was significantly less than in serum. Our results for the first time showed in Chinese population, NF against AAV2 was present in serum of all the patients with AMD or PCV and controls, and there were no significant differences among these groups. Therefore, it demonstrated there were no correlations between AAV2 NF titer and these diseases. We found NF in vitreous was considerably less than in serum in all groups. We also found no direct correlation between NF in vitreous and in serum suggesting serum antibody levels may not be used to predict their counterparts in the vitreous. Our results will provide crucial information for future clinical studies in the development of new therapies based on AAV2 mediated gene delivery in the eye.

语种: 英语
所属项目编号: 2011CB510200 ; 81100666 ; Z121100005312006
项目资助者: Genzyme Corporation of USA ; National Basic Research Program of China (973 Program) ; National Natural Science Foundation of China (NSFC) ; Research Fund for Science and Technology Program of Beijing
WOS记录号: WOS:000318604400003
Citation statistics:
内容类型: 期刊论文
URI标识: http://ir.bjmu.edu.cn/handle/400002259/61655
Appears in Collections:北京大学第二临床医学院_眼科_期刊论文

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作者单位: 1.Genzyme Corp, Sanofi Genzyme R&D Ctr, Framingham, MA 01701 USA
2.Peking Univ, Peoples Hosp, Dept Ophthalmol, Beijing 100871, Peoples R China

Recommended Citation:
Cheng, Yong,Huang, Lvzhen,Li, Xiaoxin,et al. Prevalence of Neutralizing Factors Against Adeno-Associated Virus Types 2 in Age-Related Macular Degeneration and Polypoidal Choroidal Vasculopathy[J]. CURRENT GENE THERAPY,2013,13(3):182-188.
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