Naik Rangeetha, Mukhopadhyay Arijit, Ganguli Munia
Institute of Genomics and Integrative Biology, Delhi, India.
Drug Discov Today. 2009 Mar;14(5-6):306-15. doi: 10.1016/j.drudis.2008.09.012. Epub 2008 Nov 13.
For more than two decades, gene therapy has sought to treat diseases with a genetic component. The eye is a promising target organ for gene therapy because of its unique features like easy accessibility and convenient methods of direct assessment of visual function as an effect of therapy. Several retinal diseases have been linked to specific genes in combination with environmental factors and hence gene therapy offers hope for a long-term cure for them. Developing novel non-viral routes for delivering therapeutic genes to the retina is emerging as an important area of drug delivery research. In this review, we focus on different non-viral vectors for gene delivery to the retina, the barriers that such delivery systems face and methods to overcome them.
二十多年来,基因治疗一直致力于治疗具有遗传成分的疾病。眼睛因其独特的特征,如易于接近以及作为治疗效果可直接评估视觉功能的便捷方法,而成为基因治疗的一个有前景的靶器官。几种视网膜疾病已被证明与特定基因以及环境因素有关,因此基因治疗为长期治愈这些疾病带来了希望。开发将治疗性基因递送至视网膜的新型非病毒途径正成为药物递送研究的一个重要领域。在本综述中,我们重点关注用于将基因递送至视网膜的不同非病毒载体、此类递送系统面临的障碍以及克服这些障碍的方法。