Ocular gene therapies in clinical practice: viral vectors and nonviral alternatives

被引：88
作者：
Bordet, Thierry [1 ]
Behar-Cohen, Francine [2 ,3 ,4 ,5 ]
机构：
[1] Eyevensys SAS, Paris, France
[2] INSERM, UMR S 1138, Ctr Rech Cordeliers, Team 17, Paris, France
[3] Ophtalmopole Hop Cochin, Hop Paris, AP HP, Paris, France
[4] Univ Paris 06, Sorbonne Univ, UMR S 1138, Ctr Rech Cordeliers, Paris, France
[5] Paris Descartes Univ, Sorbonne Paris Cite, UMR S 1138, Ctr Rech Cordeliers, Paris, France
来源：
DRUG DISCOVERY TODAY | 2019年 / 24卷 / 08期
关键词：
LEBER CONGENITAL AMAUROSIS; IN-VIVO; ADENOASSOCIATED VIRUS; MACULAR DEGENERATION; RETINAL DEGENERATION; DNA NANOPARTICLES; RPE65; MUTATIONS; INTRAVITREOUS INJECTION; SUBRETINAL INJECTION; LIPID NANOPARTICLES;
D O I：
10.1016/j.drudis.2019.05.038
中图分类号：
R9 [药学];
学科分类号：
1007 ;
摘要：
Ocular gene therapy has entered into clinical practice. Although viral vectors are currently the best option to replace and/or correct genes, the optimal method to deliver these treatments to the retinal pigment epithelial (RPE) cells and/or photoreceptor cells remains to be improved to increase transduction efficacy and reduce iatrogenic risks. Beyond viral-mediated gene replacement therapies, nonviral gene delivery approaches offer the promise of sustained fine-tuned expression of secreted therapeutic proteins that can be adapted to the evolving stage of the disease course and can address more common nongenetic retinal diseases, such as age-related macular degeneration (AMD). Here, we review current gene therapy strategies for ocular diseases, with a focus on clinical stage products.
引用
收藏
页码：1685 / 1693
页数：9
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