Moraru Andreea Dana, Costin Dănuț, Iorga Raluca Eugenia, Munteanu Mihnea, Moraru Radu Lucian, Branisteanu Daniel Constantin
Department of Ophthalmology, 'Grigore T. Popa' University of Medicine and Pharmacy, 700115 Iași, Romania.
Department of Ophthalmology, 'N. Oblu' Clinical Hospital, 700309 Iași, Romania.
Exp Ther Med. 2022 Jan;23(1):26. doi: 10.3892/etm.2021.10948. Epub 2021 Nov 5.
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.
眼睛被认为是基因治疗的一个有效靶点,因为它具有特殊的免疫状态,易于进行药物递送,并且受到多种遗传性疾病的影响。特别是,视网膜被考虑用于基因治疗,因为它易于可视化,没有淋巴管,外层也没有直接的血液网络,并且其细胞在出生后不再分裂,因此转基因表达不受影响。由于基因治疗目前正处于持续发展的趋势,这个新兴的基因操作技术领域已经取得了令人鼓舞的成果。这涉及到针对一些迄今为止被认为难以治疗的使人衰弱和致盲疾病的治疗方法的开发。然而,关于这些治疗方法的长期疗效和安全性,仍有许多问题未得到解答。本综述文章讨论了旨在解决与遗传性和退行性视网膜疾病相关的视力损害的基因操作技术的当前研究现状。