Suppr超能文献

视网膜疾病基因治疗的当前趋势(综述)

Current trends in gene therapy for retinal diseases (Review).

作者信息

Moraru Andreea Dana, Costin Dănuț, Iorga Raluca Eugenia, Munteanu Mihnea, Moraru Radu Lucian, Branisteanu Daniel Constantin

机构信息

Department of Ophthalmology, 'Grigore T. Popa' University of Medicine and Pharmacy, 700115 Iași, Romania.

Department of Ophthalmology, 'N. Oblu' Clinical Hospital, 700309 Iași, Romania.

出版信息

Exp Ther Med. 2022 Jan;23(1):26. doi: 10.3892/etm.2021.10948. Epub 2021 Nov 5.

Abstract

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.

摘要

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。

相似文献

1
Current trends in gene therapy for retinal diseases (Review).
Exp Ther Med. 2022 Jan;23(1):26. doi: 10.3892/etm.2021.10948. Epub 2021 Nov 5.
2
Gene therapy for inherited retinal diseases: progress and possibilities.
Clin Exp Optom. 2021 May;104(4):444-454. doi: 10.1080/08164622.2021.1880863. Epub 2021 Mar 2.
3
Gene therapy in retinal diseases: A review.
Indian J Ophthalmol. 2021 Sep;69(9):2257-2265. doi: 10.4103/ijo.IJO_3117_20.
4
Adeno-associated virus mediated gene therapy for retinal degenerative diseases.
Methods Mol Biol. 2011;807:179-218. doi: 10.1007/978-1-61779-370-7_8.
7
Recombinant AAV-mediated gene transfer to the retina: gene therapy perspectives.
Gene Ther. 2004 Oct;11 Suppl 1:S26-32. doi: 10.1038/sj.gt.3302366.
8
AAV-mediated gene therapy for retinal disorders in large animal models.
ILAR J. 2009;50(2):206-24. doi: 10.1093/ilar.50.2.206.
9
The Role of Gene Therapy in the Treatment of Retinal Diseases: A Review.
Curr Gene Ther. 2017;17(3):194-213. doi: 10.2174/1566523217666171116170040.
10
Gene therapy in inherited retinal degenerative diseases, a review.
Ophthalmic Genet. 2018 Oct;39(5):560-568. doi: 10.1080/13816810.2018.1495745. Epub 2018 Jul 24.

引用本文的文献

本文引用的文献

1
Immune function in X-linked retinoschisis subjects in an AAV8-RS1 phase I/IIa gene therapy trial.
Mol Ther. 2021 Jun 2;29(6):2030-2040. doi: 10.1016/j.ymthe.2021.02.013. Epub 2021 Feb 15.
3
Voretigene Neparvovec in Retinal Diseases: A Review of the Current Clinical Evidence.
Clin Ophthalmol. 2020 Nov 13;14:3855-3869. doi: 10.2147/OPTH.S231804. eCollection 2020.
5
AAV8-vectored suprachoroidal gene transfer produces widespread ocular transgene expression.
J Clin Invest. 2019 Aug 13;129(11):4901-4911. doi: 10.1172/JCI129085.
6
Surgical Technique for Subretinal Gene Therapy in Humans with Inherited Retinal Degeneration.
Retina. 2019 Oct;39 Suppl 1:S2-S8. doi: 10.1097/IAE.0000000000002609.
7
Suprachoroidal Triamcinolone Acetonide for Retinal Vein Occlusion: Results of the Tanzanite Study.
Ophthalmol Retina. 2018 Apr;2(4):320-328. doi: 10.1016/j.oret.2017.07.013. Epub 2017 Sep 29.
8
Luxturna: FDA documents reveal the value of a costly gene therapy.
Drug Discov Today. 2019 Apr;24(4):949-954. doi: 10.1016/j.drudis.2019.01.019. Epub 2019 Jan 31.
9
Preclinical Evaluation of ADVM-022, a Novel Gene Therapy Approach to Treating Wet Age-Related Macular Degeneration.
Mol Ther. 2019 Jan 2;27(1):118-129. doi: 10.1016/j.ymthe.2018.11.003. Epub 2018 Nov 13.
10
Gene therapy for RPE65-related retinal disease.
Ophthalmic Genet. 2018 Dec;39(6):671-677. doi: 10.1080/13816810.2018.1533027. Epub 2018 Oct 18.

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验