A review of therapeutic prospects of non-viral gene therapy in the retinal pigment epithelium.
A review of therapeutic prospects of non-viral gene therapy in the retinal pigment epithelium.
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DOI：
10.1016/j.biomaterials.2013.06.002
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发表时间：
2013-09
期刊：
影响因子：
14
通讯作者：
Naash, Muna I.
中科院分区：
文献类型：
--
作者：
Koirala, Adarsha;Conley, Shannon M.;Naash, Muna I.
近年来，眼部基因治疗作为针对角膜、视网膜和视网膜色素上皮 (RPE) 疾病的治疗途径得到了广泛的探索。腺相关病毒 (AAV) 介导的基因治疗在多项 RPE 临床试验中显示出前景，但 AAV 的有效负载能力和潜在的免疫原性有限。然而，传统上，非病毒替代品一直受到转染效率低、表达时间短和表达水平低的困扰。最近，这些缺点已开始通过使用特殊载体（如聚赖氨酸、脂质体或聚乙烯亚胺）以及通过包含合适的 DNA 元件来增强基因表达和寿命来克服。该领域的最新进展已经产生了非病毒载体，它们具有良好的安全性，缺乏免疫原性，表现出长期升高的基因表达，并在视网膜和视网膜色素上皮中显示出有效的转染，使它们准备好转向临床应用。在这里，我们讨论纳米技术和载体工程的进步，这些进步改善了非病毒基因治疗在 RPE 中的临床应用前景。
Ocular gene therapy has been extensively explored in recent years as a therapeutic avenue to target diseases of the cornea, retina and retinal pigment epithelium (RPE). Adeno-associated virus (AAV)-mediated gene therapy has shown promise in several RPE clinical trials but AAVs have limited payload capacity and potential immunogenicity. Traditionally however, non-viral alternatives have been plagued by low transfection efficiency, short-term expression and low expression levels. Recently, these drawbacks have begun to be overcome by the use of specialty carriers such as polylysine, liposomes, or polyethyleneimines, and by inclusion of suitable DNA elements to enhance gene expression and longevity. Recent advancements in the field have yielded non-viral vectors that have favorable safety profiles, lack immunogenicity, exhibit long-term elevated gene expression, and show efficient transfection in the retina and RPE, making them poised to transition to clinical applications. Here we discuss the advancements in nanotechnology and vector engineering that have improved the prospects for clinical application of non-viral gene therapy in the RPE.
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DOI：
10.1073/pnas.94.2.575
发表时间：
1997-01-21
影响因子：
11.1
作者：
Chung, JH;Bell, AC;Felsenfeld, G
通讯作者：
Felsenfeld, G
影响因子：
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作者：
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影响因子：
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通讯作者：
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影响因子：
4.8
作者：
Cai, Xue;Conley, Shannon M.;Naash, Muna I.
通讯作者：
Naash, Muna I.
影响因子：
2.6
作者：
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通讯作者：
Benham, C