The Application of gene augmentation and CRISPR/Cas9 gene editing to the treatment of neovascular fundus diseases
10.13389/j.cnki.rao.2025.0041
- VernacularTitle:基因补充与CRISPR/Cas9基因编辑在新生血管性眼底病中的应用
- Author:
Xun QIN
1
;
Zhangyu LIU
1
;
Jiayu HUANG
1
;
Jin YAO
1
Author Information
1. 210000 江苏省南京市,南京医科大学眼科医院
- Publication Type:Journal Article
- Keywords:
neovascular fundus diseases;
gene augmentation;
CRISPR/Cas9;
anti-vascular endothelial growth factor
- From:
Recent Advances in Ophthalmology
2025;45(3):233-237
- CountryChina
- Language:Chinese
-
Abstract:
Neovascular fundus diseases mainly include neovascular age-related macular degeneration(nAMD)and dia-betic retinopathy(DR).Pathological neovascular leakage and the subsequent retinal detachment are the main causes of se-vere visual impairment.Anti-vascular endothelial growth factor(VEGF)is the first-line treatment for neovascular fundus diseases,but it has shortcomings,such as the need for frequent intravitreal injections and poor patient compliance.With the annually increasing incidence of acquired neovascular fundus diseases like nAMD and DR,there is an urgent need for safer and more long-lasting treatment options.In recent years,the field of gene therapy has advanced rapidly,with thera-peutic strategies mainly involving gene supplementation and editing.The mechanism underlying gene therapy can be suc-cinctly described as the correction of pathological alterations induced by defective genes.This is achieved either by the in-troduction of exogenous functional genes to restore normal cellular processes or by directly editing aberrant genes at the ge-nomic level.Extensive basic and clinical research has demonstrated that gene therapy is both safe and effective.There are dozens of clinical trials on retinal gene therapy being carried out currently,focusing not only on inherited retinal diseases but also on neovascular fundus diseases.In this article,the application of the gene supplementation,clustered regularly in-terspaced short palindromic repeats(CRISPR)and CRISPR-associated protein 9(Cas9)system in the treatment of nAMD and DR is summarized.
