Related clinical trials and prospects of gene therapy for neovascular age-related macular degeneration

Authors: Zhang Wei,  Yan Hua
DOI: 10.3760/cma.j.cn115989-20210113-00033
Published 2021-08-10
Cite asChin J Exp Ophthalmol, 2021, 39(8): 749-754.

Abstract                              [View PDF] [Read Full Text]

Neovascular macular degeneration (nAMD) is one of the main eye diseases causing blindness in the elderly population.Early detection and early treatment are particularly important.At present, the treatment of nAMD is mainly symptomatic, including anti-vascular endothelial growth factor (VEGF) and laser treatment etc., which can not solve the problem of persistent disease activity of nAMD.Repeated treatment increases the economic burden of patients, and also the incidence of complications, such as increased intraocular pressure and inflammatory reaction.In recent years, the extensive application of gene therapy has brought more possibilities for the treatment of nAMD.It takes virus as the vector, combines the cDNA of target genes with the vector, and realizes the expression of target genes in the host cell by making use of the virus infecting host cells.Based on the successful animal experiments, phase Ⅰ and phase Ⅱ clinical trials with pigment epithelium derived factor, RNA interference, soluble VEGF receptor 1, endostatin and angiostatin, anti-VEGF Fab, soluble CD59, aflibercept, complement factor I, targets have been conducted at home and abroad.Certain achievements have been made, but some problems are also exposed.In this paper, the mechanism, research progress of clinical trials and existing problems of gene therapy for nAMD were reviewed, and the prospect of gene therapy in the future was proposed.

Key words:

Neovascular macular degeneration; Gene therapy; Clinical trial

Contributor Information

Zhang Wei

Department of Ophthalmology, Tianjin Medical University General Hospital, Tianjin 300052, China

Yan Hua

Department of Ophthalmology, Tianjin Medical University General Hospital, Tianjin 300052, China

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Updated: November 24, 2022 — 9:07 am