Advance in gene therapy of ocular disease / 中华实验眼科杂志

ABSTRACT

Currently,31 clinical trials have been approved,most of them are still in progress.Leber congenital amaurosis (LCA) has been conducted to phase Ⅲ clinical trials,the longest follow-up time was 6 years.A multicenter clinical trial about choroideremia has achieved positive effect.Retinitis pigmmentosa (RP) has been conducted to phase Ⅰ clinical trials.Gene therapy for phase Ⅰ clinical trials of age-related macular degeneration (AMD) have achieved encouraging results.RNA interference and optimized gemini surfactant-phospholipid nanoparticles(GL-NPs) have been applied to gene therapy for glaucoma and have achieved good effects.In this paper,laboratory and clinical research progress of gene therapy of LCA,RP,choroideremia and AMD,glaucoma are reviewed,including gene therapy drug delivery methods,gene carrier and common animal models,etc.Viral vectors have been widely used,the potential risk associated with immunogenicity and mutagenesis,the differences of individual reaction have promoted the exploration of a safer and more efficient method.Especially,the emergence of gene editing technology will bring a profound effect to gene therapy of eye disease field.