Current trends in gene therapy for retinal diseases (Review)

被引:12
作者
Moraru, Andreea Dana [1 ,2 ]
Costin, Danut [1 ,2 ]
Iorga, Raluca Eugenia [1 ,2 ]
Munteanu, Mihnea [3 ]
Moraru, Radu Lucian [4 ]
Branisteanu, Daniel Constantin [1 ,5 ]
机构
[1] Grigore T Popa Univ Med & Pharm, Dept Ophthalmol, 16 Univ St, Iasi 700115, Romania
[2] N Oblu Clin Hosp, Dept Ophthalmol, Iasi 700309, Romania
[3] Victor Babes Univ Med & Pharm, Dept Ophthalmol, Timioara 300041, Romania
[4] Transmed Expert Med Ctr, Dept Otorhinolaryngol, Iasi 700011, Romania
[5] Retina Ctr Eye Clin, Dept Ophthalmol, Iasi 700126, Romania
关键词
gene therapy; inherited retinal diseases; age-related macular degeneration; viral vectors; gene manipulation techniques; VIRUS; TRANSDUCTION; VECTORS; INJECTION; CELLS;
D O I
10.3892/etm.2021.10948
中图分类号
R-3 [医学研究方法]; R3 [基础医学];
学科分类号
1001 ;
摘要
The eye is considered an effective target for genetic therapy, as it has a privileged immune status, it is easily accessed for medication delivery and it is affected by a number of inherited disorders. In particular, the retina is considered for gene therapy due to the fact that it can be visualized with ease, it does not have lymphatic vessels, nor a direct blood network for the outer layers and its cells do not divide after birth, and thus transgene expression is not affected. As gene therapy is currently on a continuously progressive development trend, this emerging field of gene manipulation techniques has yielded promising results. This involves the development of treatments for a number of debilitating and blinding diseases, which were to date considered intractable. However, numerous unanswered questions remain as regards the long-term efficacy and safety profile of these treatments. The present review article discusses the current research status regarding genetic manipulation techniques aimed at addressing visual impairment related to retinal disorders, both inherited and degenerative.
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页数:6
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