Innovative gene delivery systems for retinal disease therapy  

作　　者：Hongguang Wu Ling Dong Shibo Jin Yongwang Zhao Lili Zhu 

机构地区：[1]Department of Ophthalmology,Songjiang Hospital and Songjiang Research Institute,Shanghai Key Laboratory of Emotions and Affective Disorders,Shanghai Jiao Tong University School of Medicine,Shanghai,China

出　　处：《Neural Regeneration Research》2026年第2期542-552,共11页中国神经再生研究(英文版)

基　　金：Hongguang Wu,Both authors contributed equally to this work and share first authorship;Ling Dong,Both authors contributed equally to this work and share first authorship;。

摘　　要：The human retina,a complex and highly specialized structure,includes multiple cell types that work synergistically to generate and transmit visual signals.However,genetic predisposition or age-related degeneration can lead to retinal damage that severely impairs vision or causes blindness.Treatment options for retinal diseases are limited,and there is an urgent need for innovative therapeutic strategies.Cell and gene therapies are promising because of the efficacy of delivery systems that transport therapeutic genes to targeted retinal cells.Gene delivery systems hold great promise for treating retinal diseases by enabling the targeted delivery of therapeutic genes to affected cells or by converting endogenous cells into functional ones to facilitate nerve regeneration,potentially restoring vision.This review focuses on two principal categories of gene delivery vectors used in the treatment of retinal diseases:viral and non-viral systems.Viral vectors,including lentiviruses and adeno-associated viruses,exploit the innate ability of viruses to infiltrate cells,which is followed by the introduction of therapeutic genetic material into target cells for gene correction.Lentiviruses can accommodate exogenous genes up to 8 kb in length,but their mechanism of integration into the host genome presents insertion mutation risks.Conversely,adeno-associated viruses are safer,as they exist as episomes in the nucleus,yet their limited packaging capacity constrains their application to a narrower spectrum of diseases,which necessitates the exploration of alternative delivery methods.In parallel,progress has also occurred in the development of novel non-viral delivery systems,particularly those based on liposomal technology.Manipulation of the ratios of hydrophilic and hydrophobic molecules within liposomes and the development of new lipid formulations have led to the creation of advanced non-viral vectors.These innovative systems include solid lipid nanoparticles,polymer nanoparticles,dendrimers,polymeric micelles,and polymeric

关 键 词：adeno-associated viruses delivery systems gene delivery gene therapy LENTIVIRUS nanoparticle delivery non-viral delivery retinal disease RETINA small molecular delivery 

分 类 号：R774.1[医药卫生—眼科]